EMA/CHMP (Euroopan lääkeviraston lääkevalmistekomitea)

Balversa (erdafitinib) has received a positive opinion, as monotherapy, for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.

UC is an aggressive cancer of the bladder or other parts of the urinary tract. Risk factors include smoking, exposure to toxins, and Schistosomiasis infection (mostly in Africa and the Middle East). Metastatic UC is incurable and has a poor prognosis.

Alterations in the gene encoding for fibroblast growth factor receptor (FGFR) are common in UC and thus, FGFR emerged as a novel therapeutic target for the treatment of metastatic UC. Erdafitinib is the first inhibitor targeting all isoforms of FGFR.

Eurneffy (epinephrine) has received a positive opinion for the emergency treatment of allergic reactions (anaphylaxis) due to insect stings or bites, foods, medicinal products and other allergens as well as idiopathic or exercise induced anaphylaxis. Treatment is indicated for adults and children with a body weight ≥ 30 kg.

Anaphylaxis is a severe and life-threatening allergic reaction. It requires an immediate medical intervention. Epinephrine, also known as adrenaline, counteracts the decrease in blood pressure and the obstruction of the blood vessels in the lung, which both happens during an anaphylactic reaction. There are several authorised epinephrine-containing products, which need to be injected in the case of emergency. Eurneffy is the first nasal epinephrine spray which has been authorised. The EMA has published a press release on Eurneffy.

mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine) has received a positive opinion for the active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus in adults 60 years of age and older.

Respiratory Syncytial Virus (RSV) is a single-stranded RNA virus that causes the majority of cases of respiratory hospitalisation in infants. In the elderly population, approximately 15% of acute respiratory infections due to RSV lead to hospitalisation. Lower respiratory tract disease is characterised by bronchiolitis and pneumonia, and is potentially life-threatening. mResvia is a mRNA-based vaccine that encodes for RSV glycoprotein F. This protein is the target of neutralising antibodies that mediate protection against RSV-associated respiratory tract disease.

Ordspono (odronextamab) has received a positive opinion for a conditional marketing authorisation (CMA) for the following indications:

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (r/r FL) after two or more lines of systemic therapy.

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) after two or more lines of systemic therapy.

Follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) are two subtypes of non-Hodgkin lymphoma (NHL), a heterogeneous group of lymphoid malignancies deriving from B-cells or T-cells. FL and DLBCL are B-cell lymphomas.

Like many mature B-cell lymphomas, FL and DLBCL express the surface antigen CD20. Odronextamab is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Odronextamab binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated tumour cell lysis.

Piasky (crovalimab) has received a positive opinion, as monotherapy, for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with paroxysmal nocturnal haemoglobinuria (PNH):

• In patients with haemolysis with clinical symptom(s) indicative of high disease activity.

• In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.

PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.

Crovalimab is a monoclonal antibody targeting the complement protein C5, thereby protecting it from cleavage. Thus, the complement system initiation is blocked and intravascular haemolysis is reduced.

Tauvid (flortaucipir (¹⁸F)) has received a positive opinion for the following indication:

This medicinal product is for diagnostic use only.

Flortaucipir (¹⁸F) is a radiopharmaceutical indicated for positron emission tomography (PET) imaging of the brain to assess the neocortical distribution of aggregated tau neurofibrillary tangles (NFTs) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD). Flortaucipir (¹⁸F) is an adjunct to clinical and other diagnostic evaluations.

PET imaging is a diagnostic procedure which is used, e.g., for the detection of Alzheimer’s disease. To this end, certain Alzheimer-specific structures in the brain need to be labelled with radioactive, positron-emitting molecules. Tauvid contains radioactive fluor (¹⁸F) and binds to these structures, enabling the diagnostic evaluation.

Winrevair (sotatercept) has received a positive opinion, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity.

Pulmonary arterial hypertension (PAH) is high blood pressure in the blood vessels which lead from the heart to the lung. PAH is a rare condition and is associated with difficulty to breathe, chest pain and dizziness. As the heart needs to work harder to pump blood into the lung, it may eventually be affected. PAH can be a life-threatening disease.

Sotatercept is a recombinant protein and an activin inhibitor. Activin is overexpressed in PAH and promotes the growth of new blood vessels in the lungs, which results in narrowing and thickening of vessel walls. The treatment with sotatercept is expected to improve exercise capacity of patients. Winrevair is a first-in-class product and the EMA has published a press release.

Balversa (erdafitinib) has received a positive opinion, as monotherapy, for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.

UC is an aggressive cancer of the bladder or other parts of the urinary tract. Risk factors include smoking, exposure to toxins, and Schistosomiasis infection (mostly in Africa and the Middle East). Metastatic UC is incurable and has a poor prognosis.

Alterations in the gene encoding for fibroblast growth factor receptor (FGFR) are common in UC and thus, FGFR emerged as a novel therapeutic target for the treatment of metastatic UC. Erdafitinib is the first inhibitor targeting all isoforms of FGFR.

Eurneffy (epinephrine) has received a positive opinion for the emergency treatment of allergic reactions (anaphylaxis) due to insect stings or bites, foods, medicinal products and other allergens as well as idiopathic or exercise induced anaphylaxis. Treatment is indicated for adults and children with a body weight ≥ 30 kg.

Anaphylaxis is a severe and life-threatening allergic reaction. It requires an immediate medical intervention. Epinephrine, also known as adrenaline, counteracts the decrease in blood pressure and the obstruction of the blood vessels in the lung, which both happens during an anaphylactic reaction. There are several authorised epinephrine-containing products, which need to be injected in the case of emergency. Eurneffy is the first nasal epinephrine spray which has been authorised. The EMA has published a press release on Eurneffy.

mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine) has received a positive opinion for the active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus in adults 60 years of age and older.

Respiratory Syncytial Virus (RSV) is a single-stranded RNA virus that causes the majority of cases of respiratory hospitalisation in infants. In the elderly population, approximately 15% of acute respiratory infections due to RSV lead to hospitalisation. Lower respiratory tract disease is characterised by bronchiolitis and pneumonia, and is potentially life-threatening. mResvia is a mRNA-based vaccine that encodes for RSV glycoprotein F. This protein is the target of neutralising antibodies that mediate protection against RSV-associated respiratory tract disease.

Ordspono (odronextamab) has received a positive opinion for a conditional marketing authorisation (CMA) for the following indications:

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (r/r FL) after two or more lines of systemic therapy.

Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) after two or more lines of systemic therapy.

Follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) are two subtypes of non-Hodgkin lymphoma (NHL), a heterogeneous group of lymphoid malignancies deriving from B-cells or T-cells. FL and DLBCL are B-cell lymphomas.

Like many mature B-cell lymphomas, FL and DLBCL express the surface antigen CD20. Odronextamab is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Odronextamab binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated tumour cell lysis.

Piasky (crovalimab) has received a positive opinion, as monotherapy, for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with paroxysmal nocturnal haemoglobinuria (PNH):

• In patients with haemolysis with clinical symptom(s) indicative of high disease activity.

• In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.

PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.

Crovalimab is a monoclonal antibody targeting the complement protein C5, thereby protecting it from cleavage. Thus, the complement system initiation is blocked and intravascular haemolysis is reduced.

Tauvid (flortaucipir (¹⁸F)) has received a positive opinion for the following indication:

This medicinal product is for diagnostic use only.

Flortaucipir (¹⁸F) is a radiopharmaceutical indicated for positron emission tomography (PET) imaging of the brain to assess the neocortical distribution of aggregated tau neurofibrillary tangles (NFTs) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD). Flortaucipir (¹⁸F) is an adjunct to clinical and other diagnostic evaluations.

PET imaging is a diagnostic procedure which is used, e.g., for the detection of Alzheimer’s disease. To this end, certain Alzheimer-specific structures in the brain need to be labelled with radioactive, positron-emitting molecules. Tauvid contains radioactive fluor (¹⁸F) and binds to these structures, enabling the diagnostic evaluation.

Winrevair (sotatercept) has received a positive opinion, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity.

Pulmonary arterial hypertension (PAH) is high blood pressure in the blood vessels which lead from the heart to the lung. PAH is a rare condition and is associated with difficulty to breathe, chest pain and dizziness. As the heart needs to work harder to pump blood into the lung, it may eventually be affected. PAH can be a life-threatening disease.

Sotatercept is a recombinant protein and an activin inhibitor. Activin is overexpressed in PAH and promotes the growth of new blood vessels in the lungs, which results in narrowing and thickening of vessel walls. The treatment with sotatercept is expected to improve exercise capacity of patients. Winrevair is a first-in-class product and the EMA has published a press release.

Betmiga (mirabegron): extension of indication to include the treatment of neurogenic detrusor overactivity (NDO) in paediatric patients aged 3 to less than 18 years.

Betmiga is already authorised for adults with overactive bladder syndrome.

Beyfortus (nirsevimab): extension of indication to include the prevention of Respiratory Syncytial Virus (RSV) lower respiratory tract disease in children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

Beyfortus is already authorised for neonates and infants during their first RSV season.

Cresemba (isavuconazole): extension of indication to include the treatment of patients from 1 year of age and older (pharmaceutical form: powder for concentrate for solution for infusion) and of adults and paediatric patients from 6 years of age (pharmaceutical form: hard capsules) for the treatment of

• invasive aspergillosis

• mucormycosis in patients for whom amphotericin B is inappropriate

Cresemba is already authorised for the treatment of both infections in adults, as powder for concentrate for solution for infusion.

Imcivree (setmelanotide): extension of indication to include the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS), loss-of-function biallelic proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above.

Imcivree is already authorised in children from 6 years of age.

Imfinzi (durvalumab): extension of indication to include the following indication:

Imfinzi in combination with carboplatin and paclitaxel is indicated for the first-line treatment of adults with primary advanced or recurrent endometrial cancer who are candidates for systemic therapy, followed by maintenance treatment with:

• Imfinzi as monotherapy in endometrial cancer that is mismatch repair deficient (dMMR)

• Imfinzi in combination with olaparib in endometrial cancer that is mismatch repair proficient (pMMR).

Imfinzi is already authorised for non-small cell lung cancer, small cell lung cancer, biliary tract cancer and hepatocellular carcinoma.

Infanrix hexa (diphtheria (D), tetanus (T), pertussis (acellular, component) (Pa), hepatitis B (rDNA) (HBV), poliomyelitis (inactivated) (IPV) and Haemophilus influenzae type b (Hib) conjugate vaccine (adsorbed)): extension of indication to include the use as primary and booster vaccination of infants from the age of 6 weeks and toddlers against diphtheria, tetanus, pertussis, hepatitis B, poliomyelitis and disease caused by Haemophilus influenzae type b.

Infanrix hexa was already authorised without a specified lower age limit.

Lynparza (olaparib): extension of indication to include, in combination with durvalumab, the maintenance treatment of adult patients with primary advanced or recurrent endometrial cancer that is mismatch repair proficient (pMMR) whose disease has not progressed on first-line treatment with durvalumab in combination with carboplatin and paclitaxel.

Lynparza is already authorised for ovarian cancer, breast cancer, adenocarcinoma of the pancreas and prostate cancer.

Pegasys (peginterferon alfa-2a): extension of indication to include the following indications:

• Pegasys is indicated as monotherapy in adults for the treatment of polycythaemia vera.
• Pegasys is indicated as monotherapy in adults for the treatment of essential thrombocythaemia.

Pegasys is already authorised for the treatment of chronic hepatitis B.

Tepkinly (epcoritamab): extension of indication to include the monotherapy treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

Tepkinly is already authorised for the treatment of diffuse large B-cell lymphoma (DLBCL).

Vabysmo (faricimab): extension of indication to include the treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) in adult patients.

Vabysmo is already authorised for neovascular (wet) age-related macular degeneration and visual impairment due to diabetic macular oedema.

Xalkori (crizotinib): extension of indication to include the following indications:

The treatment of paediatric patients (age ≥1 to <6 years) with relapsed or refractory systemic anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALCL)

The treatment of paediatric patients (age ≥1 to <6 years) with recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT)

Xalkori is already authorised in children from ≥6 to <18 years in the same indications as listed above, as well as in adults with ALK- and ROS1-positive non-small cell lung cancer (NSCLC).

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Qalsody (tofersen) is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

EPAR Qalsody.

Fabhalta (iptacopan) is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

EPAR Fabhalta.

Datopotamab - Treatment of adult patients with inoperable or metastatic HR-positive / HER2-negative breast cancer with disease progression following chemotherapy in the metastatic setting

Datopotamab - Treatment of adult patients with locally advanced or metastatic non squamous non-small cell lung cancer (NSCLC)

Resminostat - Treatment of patients with advanced stage mycosis fungoides (MF) and Sézary syndrome (SS)

Seladelpar lysine dihydrate - Treatment of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child-Pugh A) in combination with ursodeoxycholic acid (UDCA) who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA

Nirogacestat - Treatment of desmoid tumours

Resmetirom - Treatment of adults with non-alcoholic steatohepatitis (NASH)/metabolic dysfunctionassociated steatohepatitis (MASH) with liver fibrosis

 

 

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation

https://www.ema.europa.eu/en/news/first-nasal-adrenaline-spray-emergency-treatment-against-allergic-reactions

https://www.ema.europa.eu/en/news/positive-chmp-opinion-first-class-medicine-treat-pulmonary-arterial-hypertension

https://www.ema.europa.eu/en/documents/assessment-report/qalsody-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/fabhalta-public-assessment-report_en.pdf

Adzynma (rADAMTS13) has received a positive opinion for a marketing authorisation under exceptional circumstances, as a replacement therapy, for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).
cTTP is a rare disorder of the small blood vessels caused by mutations in the ADAMTS13 gene. It is inherited in an autosomal recessive order. ADAMTS13 is an antithrombocytic enzyme, and its absence leads to a reduced number of platelets in the blood, while the risk for thrombosis is increased. Patients may develop neurological, renal, cardiac and gastrointestinal symptoms due to widespread microvascular thrombosis.
Adzynma is a recombinant protein and is intended to replace the missing ADAMTS13 gene product.

Akantior (polihexanide) has received a positive opinion for the treatment of Acanthamoeba keratitis in adults and children from 12 years of age.
Acanthamoeba keratitis is a rare eye disease which primarily affects contact lens wearers. The infectious disease is caused by acanthamoeba, a certain amoeba species, which can colonise the human eye as a parasite. Symptoms may include pain, decreased vision, light sensitivity and tearing. 
Polihexanide, the active substance in Akantior, destroys both the cell membrane and the chromosomes of acanthamoeba. It is administered as an eye drops solution.

Cejemly (sugemalimab) has received a positive opinion for the first-line treatment, in combination with platinum-based chemotherapy, of adults with metastatic non-small-cell lung cancer (NSCLC) with no sensitising EGFR mutations, or ALK, ROS1 or RET genomic tumour aberrations.
NSCLC is a serious and often fatal disease that accounts for 80 to 85% of all lung cancers. A significant number of patients present with driver mutations in oncogenes, some of which can be aimed at by targeted therapies. Patients without known driver mutations need broader-acting therapies.
Sugemalimab is a checkpoint inhibitor, targeting PD-L1 (programmed cell death protein 1) on cancer cells. The corresponding receptor, PD-1, is expressed on immune cells. Since activation of PD-1 down-regulates the immune system, blocking of the interaction between PD-1 and PD-L1 enhances the T cell responses against the cancer cells.

Durveqtix (fidanacogene elaparvovec) has received a positive opinion for a conditional marketing authorisation for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74.
Congenital haemophilia is an X-linked recessive disease caused by mutations in the genes encoding for the coagulation factor VIII (haemophilia A) or IX (haemophilia B). It is characterised by the inability to form blood clots, which results in a higher risk of bruising, internal bleeding and bleeding inside the joints. The disease can be classified as mild, moderate or severe, depending on the endogenous plasma activity levels of clotting factors. Patients who are treated with anti-haemophilic factors to replace the deficient coagulation factor can develop anti-factor VIII or XI alloantibodies (inhibitors), which neutralise the activity of the administered replacement factors.
Fidanacogene elaparvovec, the active substance of Durveqtix, is a gene therapy that aims at enabling the body to produce factor IX itself and prevent and control bleeding. While many of the authorised products for the treatment of haemophilia B require frequent infusions, Durveqtix needs to be administered only once.
Durveqtix was supported through EMA‘s Priority Medicines (PRIME) scheme. The EMA has published a press release on Durveqtix.

Fluenz (influenza vaccine (live attenuated, nasal)) has received a positive opinion for the prevention of influenza in children and adolescents from 24 months to less than 18 years of age.
Influenza is an infectious disease of the nose, throat and lungs which is caused by the influenza virus. Infection usually occurs by droplet spread from infected people to uninfected people through inhalation. The viral infection can cause fever, cough and breathing problems up to pneumonia in humans.
Fluenz is a live attenuated influenza vaccine. It will be available as a nasal spray. 

GalliaPharm (Germanium (68Ge) chloride / Gallium (68Ga) chloride) has received a positive opinion for in vitro radiolabelling of various kits for radiopharmaceutical preparation developed and approved for radiolabelling with such eluate, to be used for positron emission tomography (PET) imaging.
PET imaging is a diagnostic procedure which is used, e.g., for several types of cancer. To this end, carrier products need to be labelled with radioactive, positron-emitting molecules like gallium (68Ga).
GalliaPharm contains radioactive Germanium (68Ge) which is used to generate a 68Ga solution for labelling. It is not intended for direct use in patients.

Ixchiq (Chikungunya vaccine (live)) has received a positive opinion for active immunisation for the prevention of disease caused by chikungunya virus (CHIKV) in individuals 18 years and older. The use of this vaccine should be in accordance with official recommendations.
Chikungunya is a viral disease caused by the Chikungunya virus, which is transmitted to humans by infected mosquitoes. Symptoms include fever and severe joint pain, muscle pain, headache nausea and fatigue. Chikungunya is a debilitating disease and can lead to multiorgan failure in a small proportion of patients.
Ixchiq is the first vaccine in the EU to protect adults from Chikungunya infection. It was supported through EMA‘s Priority Medicines (PRIME) scheme and reviewed under the OPEN (Opening procedures at EMA to non-EU authorities) framework to promote global public health. 
The EMA has published a press release on Ixchiq.

Zegalogue (dasiglucagon) has received a positive opinion for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 6 years and over with diabetes mellitus.
There are two types of diabetes mellitus. Type 1 diabetes is an autoimmune disease in which pancreatic cells are destroyed by the immune system, leading to a decreased insulin production. Type 2 diabetes is a metabolic disease characterised by high blood sugar and insulin resistance and it accounts for around 90% of diabetes cases. Type 2 diabetes occurs primarily as a result of obesity and sedentary lifestyle, and the prevalence increases globally along with obesity and overweight.
In patients with diabetes mellitus, insulin treatment can sometimes lead to hypoglycaemia, which is a too low glucose level in the blood.
Dasiglucagon, the active substance in Zegalogue, acts on a receptor relevant for glucose metabolism and is intended to increase plasma glucose levels. It is administered as subcutaneous injection.

Dupixent (dupilumab): extension of indication to include, as add-on maintenance treatment in adults, the treatment of uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils on a combination of an inhaled corticosteroid (ICS), a long-acting beta2-agonist (LABA), and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate.

Dupixent is already authorised for atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, prurigo nodularis, and eosinophilic esophagitis.

Eliquis (apixaban): extension of indication to include the treatment of venous thromboembolism (VTE) and prevention of recurrent VTE in paediatric patients from 28 days to less than 18 years of age.

Eliquis is already authorised for the prevention of VTE in adults, as well as the prevention of stroke and systemic embolism, the treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), and prevention of recurrent DVT and PE in adults.

Kinpeygo (budesonide): extension of indication to include the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g).

Kinpeygo is already authorised for IgAN patients with worse urine protein-to-creatinine ratios.

Livmarli (maralixibat): extension of indication to include the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients 3 months of age and older.

Livmarli is already authorised for the treatment of cholestatic pruritus in patients with Alagille syndrome.

Skyrizi (risankizumab): extension of indication to include the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy.

Skyrizi is already authorised for the treatment of plaque psoriasis, psoriatic arthritis and Crohn’s disease.

Tagrisso (osimertinib): extension of indication to include, in combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adult patients with advanced NSCLC whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations.

Tagrisso is already authorised for different treatment settings in non-small cell lung cancer (NSCLC).

Tevimbra (tislelizumab): extension of indication to include several treatment settings of non-small cell lung cancer.

1. Tevimbra in combination with pemetrexed and platinum-containing chemotherapy is indicated for the first-line treatment of adult patients with non-squamous NSCLC whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have:

• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or

• metastatic NSCLC.

2. Tevimbra in combination with carboplatin and either paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous NSCLC who have:

• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or

• metastatic NSCLC.

3. Tevimbra as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.

Tevimbra is already authorised for the treatment of oesophageal squamous cell carcinoma.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Ryzneuta (efbemalenograstim alfa) is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).

EPAR Ryzneuta.

Celldemic (zoonotic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures)) is indicated for active immunisation against H5N1 subtype of Influenza A virus in adults and infants from 6 months of age and above.

EPAR Celldemic.

Filspari (sparsentan) is indicated for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥ 0.75 g/g, see section 5.1 in the Summary of Product Characteristics).

EPAR Filspari.

Incellipan (pandemic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures) is indicated for active immunisation against influenza in an officially declared pandemic.

EPAR Incellipan.

Tizveni (tislelizumab) is indicated for the following indications of non-small cell lung cancer:

1. Tizveni in combination with pemetrexed and platinum-containing chemotherapy is indicated for the first-line treatment of adult patients with non-squamous non-small cell lung cancer whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have:

• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or

• metastatic NSCLC

2. Tizveni in combination with carboplatin and either paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous non-small cell lung cancer who have:

• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or

• metastatic NSCLC.

3. Tizveni as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.

EPAR Tizveni.

Voydeya (danicopan) is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.

EPAR Voydeya.

Zynyz (retifanlimab) is indicated as monotherapy for the first-line treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) not amenable to curative surgery or radiation therapy.

EPAR Zynyz.

Awiqli (insulin icodec) is indicated for the treatment of diabetes mellitus in adults.

EPAR Awiqli.

Emblaveo (aztreonam / avibactam) is indicated for the treatment of the following infections in adult patients:

• Complicated intra-abdominal infection (cIAI)

• Hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP)

• Complicated urinary tract infection (cUTI), including pyelonephritis

Emblaveo is also indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options.

EPAR Emblaveo.

Lytenava (bevacizumab gamma) is indicated in adults for treatment of neovascular (wet) age-related macular degeneration (nAMD).

EPAR Lytenava.

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines

https://www.ema.europa.eu/en/partners-networks/international-activities/multilateral-coalitions-initiatives/opening-procedures-ema-non-eu-authorities-open-initiative

https://www.ema.europa.eu/en/glossary/exceptional-circumstances

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/accelerated-assessment

https://www.ema.europa.eu/en/news/new-gene-therapy-treatment-haemophilia-b

https://www.ema.europa.eu/en/news/first-vaccine-protect-adults-chikungunya

https://www.ema.europa.eu/en/documents/assessment-report/ryzneuta-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/celldemic-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/filspari-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/incellipan-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/tizveni-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/voydeya-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/zynyz-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/awiqli-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/emblaveo-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/en/documents/assessment-report/lytenava-epar-public-assessment-report_en.pdf

Altuvoct (efanesoctocog alfa) has received a positive opinion for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).
Congenital haemophilia is an X-linked recessive disease caused by mutations in the genes coding for the coagulation factor VIII (haemophilia A) or IX (haemophilia B). It is characterised by the inability to form blood clots, which results in a higher risk of bruising, internal bleeding and bleeding inside the joints. The disease can be classified as mild, moderate or severe, depending on the endogenous plasma activity levels of clotting factors. Patients who are treated with anti-haemophilic factors to replace the deficient coagulation factor can develop anti-factor VIII or XI alloantibodies (inhibitors), which neutralise the activity of the administered replacement factors. 
Efanesoctocog alfa is a long-acting version of recombinant factor VIII (FVIII) for the control and prevention of bleeding episodes in individuals with haemophilia A. It is a genetically engineered fusion protein of FVIII, von-Willebrand factor (VWF) and a peptide which prevents the fusion protein from being degraded, resulting in a longer half-life.

Fruzaqla (fruquintinib) has received a positive opinion for the monotherapy treatment of adult patients with metastatic colorectal cancer (mCRC) who have been previously treated with available standard therapies, including fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapies, anti-VEGF agents, and anti-EGFR agents, and who have progressed on or are intolerant to treatment with either trifluridine-tipiracil or regorafenib.
Colorectal cancer (CRC) is a common and lethal disease. CRC can be diagnosed after the onset of symptoms, like abdominal pain or bleeding, or through routine screening of asymptomatic individuals. CRC at metastatic stage has a poor prognosis, with a five-year survival rate of less than 10%.
Fruquintinib is an orally available inhibitor of vascular endothelial growth factor receptors (VEGFRs) 1, 2, and 3. Binding of VEGF to its receptor promotes angiogenesis, the formation of new blood vessels. This is a mechanism cancer cells can take advantage of, as they can be supplied with oxygen and nutrients. Via upregulation of VEGFR, continued growth in solid tumours can be enabled. 

Jeraygo (aprocitentan) has received a positive opinion for the treatment of resistant hypertension in adult patients in combination with at least three antihypertensive medicinal products.
Hypertension, or high blood pressure, is a leading cause of cardiovascular disease and mortality. About 10% of patients have a difficult-to-control hypertension which means that blood pressure cannot be controlled despite being treated with at least three antihypertensive drugs.
Aprocitentan is an orally available endothelin receptor antagonist. Binding of aprocitentan to the receptor leads to relaxation of small blood vessels thus preventing a rise in blood pressure.

Obgemsa (vibegron) has received a positive opinion for the symptomatic treatment of adult patients with overactive bladder (OAB) syndrome.
Overactive bladder (OAB) syndrome is a highly-prevalent disease among middle aged and elderly adults. It is estimated that one third of men and women 75 years of age and older are affected. It is characterised by urinary urgency and frequency, with or without the involuntary loss of urine.
Vibegron is a beta-3 adrenergic receptor agonist. Binding of vibegron to the receptor leads to relaxation of the smooth muscle cells of the bladder, thus increasing bladder capacity.

Truqap (capivasertib) has received a positive opinion, in combination with fulvestrant, for the treatment of adult patients with oestrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations following recurrence or progression on or after an endocrine-based regimen.
Breast cancer (BC) is the leading cause of cancer and cancer deaths in women worldwide, especially when diagnosed at an advanced stage. BC is categorised to subtypes according to its histopathology. Oestrogen receptor (ER) and progesterone receptor (PR) positive tumours are referred to as hormone-receptor (HR) positive BC. About 70% of all diagnosed BCs are HR positive and HER2 negative.
Capivasertib is a selective inhibitor of AKT 1, 2 and 3. The AKT serine/threonine protein kinases are key downstream effectors of the PI3K/AKT/PTEN pathway, promoting cell proliferation, survival, metabolism, and gene expression. Several other AKT inhibitors are currently under development for different oncological conditions.
 

 

Alecensa (alectinib): extension of indication to include, as monotherapy, the adjuvant treatment following complete tumour resection for adult patients with ALK-positive non-small cell lung cancer (NSCLC) at high risk of recurrence.
Alecensa is already authorised for different, non-adjuvant treatment settings of NSCLC. 

Opdivo (nivolumab): extension of indication to include, in combination with cisplatin and gemcitabine, the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma.
Opdivo is already authorised for a number of solid tumours, including the second-line treatment of urothelial carcinoma. 

Rozlytrek (entrectinib): extension of indication to include the treatment of adult and paediatric patients older than 1 month with solid tumours that have a NTRK gene fusion,
• who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and
• who have not received a prior NTRK inhibitor
• who have no satisfactory treatment options
Rozlytrek is a tumour-agnostic cancer treatment and has previously been authorised for the treatment of adult and adolescent patients 12 years of age and older with solid, NTRK gene fusion expressing tumours, as well as for the treatment of adult patients with ROS1-positive, advanced non-small cell lung cancer. 

Rybrevant (amivantamab): extension of indication to include, in combination with carboplatin and pemetrexed, the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating EGFR Exon 20 insertion mutations.
Rybrevant is already authorised for the second-line treatment of EGFR mutated NSCLC. 

Sirturo (bedaquiline): extension of indication to include the use as part of an appropriate combination regimen in adult and paediatric patients (5 years to less than 18 years of age and weighing at least 15 kg) with pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampicin and isoniazid.
Sirturo is already authorised for a different treatment setting in pulmonary TB.

Triumeq (dolutegravir / abacavir / lamivudine): extension of indication to include the treatment of Human Immunodeficiency Virus type 1 (HIV-1) infected children of at least 3 months of age weighing at least 6 kg to less than 25 kg.
Up to now, Triumeq (as dispersible tablet) was authorised for children weighing at least 14 kg, without age restriction. In addition, the separate film-coated tablet formulation remains authorised for children, adolescents and adults weighing at least 25 kg.
 

-    Acoramidis – Orphan - for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
-    Diflunisal – Orphan - Treatment of ATTR amyloidosis.
-    Lazertinib - treatment of adult patients with advanced non-small cell lung cancer (NSCLC).
-    Linvoseltamab - monotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma.
-    Nemolizumab - for the treatment of moderate-to-severe atopic dermatitis and for the treatment of prurigo nodularis.
-    Pegfilgrastim – paediatric use marketing authorisation (PUMA) - treatment of neutropenia in paediatric patients.
-    Tisotumab vedotin - treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy.
-    Human albumin solution - vitrification of human MII-phase oocytes and embryos for assisted reproductive technology (ART).

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context
 

Awiqli (insulin icodec) received a positive opinion for the treatment of diabetes mellitus in adults.
Awiqli was developed for the treatment of both type 1 and type 2 diabetes mellitus. Type 1 diabetes is an autoimmune disease in which pancreatic cells are destroyed by the immune system, leading to a decreased insulin production. Type 2 diabetes is a metabolic disease characterised by high blood sugar and insulin resistance and it accounts for around 90% of diabetes cases. Type 2 diabetes occurs primarily as a result of obesity and sedentary lifestyle, and the prevalence increases globally along with obesity and overweight.
The active substance of Awiqli, insulin icodec, is a new, very long-acting human insulin analogue that needs to be injected only once weekly.

Emblaveo (aztreonam / avibactam) received a positive opinion for the treatment of the following infections in adult patients:
• Complicated intra-abdominal infection (cIAI)
• Hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP)
• Complicated urinary tract infection (cUTI), including pyelonephritis
Emblaveo is also indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options.
Emblaveo combines two antibiotics. Aztreonam is a monocyclic beta-lactam agent (monobactam) that inhibits bacterial cell wall synthesis by targeting penicillin-binding proteins. Avibactam is a beta-lactamase inhibitor that prevents a certain class of bacterial enzymes, beta-lactamases, from hydrolysing aztreonam. The combination of these two compounds aims at multidrug-resistant, Gram-negative bacteria, which are estimated to cause 35,000 deaths in the EU every year.
Emblaveo underwent accelerated assessment, recognising that the product is of major interest for public health. The EMA has published a press release on Emblaveo.

Fabhalta (iptacopan) received a positive opinion for the treatment, as monotherapy, of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.
PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.
Iptacopan is an orally available inhibitor of the alternative complement pathway, and can thus control both extravascular and intravascular haemolysis. The development of Fabhalta was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support.
The EMA has published a press release on Fabhalta.

Lytenava (bevacizumab gamma) received a positive opinion for the treatment of adult patients with neovascular (wet) age-related macular degeneration (nAMD).
Vascular endothelial growth factor (VEGF) is the main protein responsible for the induction of blood vessel growth and, therefore, it is thought contribute to the pathophysiology of retinal vascular diseases, such as nAMD. Bevacizumab is a monoclonal antibody that targets VEGF, thereby blocking the binding to its receptor on endothelial cells, and preventing the growth of new abnormal blood vessels in the back of the eye.
 

Bimzelx (bimekizumab): extension of indication to include the treatment of active moderate to severe hidradenitis suppurativa (HS, acne inversa) in adults with an inadequate response to conventional systemic HS therapy.
Bimzelx is already authorised for the treatment of plaque psoriasis, psoriatic arthritis, and axial spondyloarthritis. 

Nilemdo (bempedoic acid): extension of indication to include the treatment of adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:
• in patients on a maximum tolerated dose of a statin with or without ezetimibe or,
• alone or in combination with ezetimibe in patients who are statin-intolerant, or for whom a statin is contraindicated.
Nilemdo is already authorised for the treatment of hypercholesterolaemia and mixed dyslipidaemia. 

Nustendi (bempedoic acid / ezetimibe): extension of indication to include the treatment of adults with established risk of, or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:
• in patients on a maximum tolerated dose of a statin and not adequately controlled with additional ezetimibe treatment or,
• in patients who are either statin-intolerant, or for whom a statin is contraindicated, and not adequately controlled with ezetimibe treatment or,
• in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets.
Nustendi is already authorised for the treatment of hypercholesteraemia and mixed dyslipidaemia. 

Onivyde pegylated liposomal (irinotecan hydrochloride trihydrate): extension of indication to include, in combination with oxaliplatin, 5-fluorouracil (5-FU) and leucovorin (LV), the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas.
Onivyde pegylated liposomal is already authorised in a second-line setting for pancreatic cancer, in combination with 5-FU and LV. 

Retsevmo (selpercatinib): extension of indication to include the treatment of adult patients with advanced RET fusion-positive solid tumours, when treatment options not targeting RET provide limited clinical benefit, or have been exhausted.
Retsevmo is already authorised, based on a conditional marketing authorisation, for the treatment of RET fusion positive non-small cell lung cancer and thyroid cancer. 

Xtandi (enzalutamide): extension of indication to include, as monotherapy or in combination with androgen deprivation therapy, the treatment of adult men with high risk biochemical recurrent (BCR) non-metastatic hormone sensitive prostate cancer (nmHSPC) who are unsuitable for salvage radiotherapy.
Xtandi is already authorised for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy, and for various subtypes of castration-resistant prostate cancer (CRPC). 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage: 

Krazati (adagrasib) as monotherapy is indicated for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and disease progression after at least one prior systemic therapy.
EPAR Krazati.

Rystiggo (rozanolixizumab) is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
EPAR Rystiggo.

Casgevy (exagamglogene autotemcel) is indicated for the following indications:
β-thalassemia
Casgevy is indicated for the treatment of transfusion-dependent β-thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.
Sickle cell disease
Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.
EPAR Casgevy.

Skyclarys (omaveloxolone) is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
EPAR Skyclarys.

Exblifep (cefepime / enmetazobactam) is indicated for the treatment of the following infections in adults:
• Complicated urinary tract infections (cUTI), including pyelonephritis
• Hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP),
and for the treatment of patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above.
EPAR Exblifep.
 

-    Garadacimab – Orphan – Routine prevention of attacks of hereditary angioedema (HAE)
-    Chikungunya virus, strain CHIKV LR2006-OPY1, live attenuated – Accelerated review – Prevention of disease caused by chikungunya (CHIKV) virus 
-    Beremagene geperpavec – Orphan – ATMP – Treatment of patients from birth with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene
-    Repotrectinib – Treatment of ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) and for solid tumours
-    Autologous cartilage-derived articular chondrocytes, in-vitro expanded – ATMP – Repair of symptomatic, localised, full-thickness cartilage defects of the knee joint grade III or IV
-    Govorestat – Orphan – treatment of adults and children aged 2 years and older with a confirmed diagnosis of classic galactosemia
-    Odevixibat – Treatment of cholestatic pruritus in Alagille syndrome (ALGS)
-    Vorasidenib – Orphan – Accelerated review – Treatment of predominantly non-enhancing astrocytoma or oligodendroglioma with a IDH1 R132 mutation or IDH2 R172 mutation
-    Belzutifan – Treatment of adult patients with advanced renal cell carcinoma (RCC) and treatment of adult patients with von Hippel-Lindau (VHL) disease

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/accelerated-assessment
https://www.ema.europa.eu/en/news/new-antibiotic-fight-infections-caused-multidrug-resistant-bacteria
https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines
https://www.ema.europa.eu/en/news/first-oral-monotherapy-patients-paroxysmal-nocturnal-haemoglobinuria

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation

https://www.ema.europa.eu/en/documents/assessment-report/krazati-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/rystiggo-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/casgevy-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/skyclarys-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/exblifep-epar-public-assessment-report_en.pdf
 

Two products for the active immunisation against H5N1 subtype of influenza A virus received a positive opinion this month:
Celldemic (zoonotic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures)) received a positive opinion for the active immunisation against H5N1 subtype of Influenza A virus in adults and infants from 6 months of age and above.
Incellipan (pandemic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures) received a positive opinion for the active immunisation against influenza in an officially declared pandemic.
Both products are identical, but are intended to target different applications (zoonotic vs. pandemic vaccine).
Influenza subtype H5N1 is a zoonotic virus, which primarily occurs in birds (“bird flu”) and can be transmitted to humans. Pandemic influenza outbreaks can occur when a new highly infectious virus strain enters a population with low immunity from previous exposure. Infection with influenza virus usually occurs by droplet spread from infected people to uninfected people through inhalation. The viral infection can cause fever, cough and breathing problems up to pneumonia in humans. Although human transmission of H5N1 is rare, the mortality rate from H5N1 infection is high.

Filspari (sparsentan) received a positive opinion, recommending the granting of a conditional marketing authorisation, for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion >1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g).
IgAN, also known as Berger’s disease, is a progressive autoimmune glomerulonephritis characterised by galactose-deficient IgA1 (GdIgA1) deposits within the glomeruli of the kidney. This accumulation triggers inflammatory events, ultimately causing irreversible scarring of the glomeruli (glomerulosclerosis) and loss of filtration capacity. The primary form of the disease is characterised by the lack of relevant associated co-morbidities. Although the aetiology is unknown, it has been shown that the mucosal immune system (particularly of the gastrointestinal tract) is involved in the development of the disease.
Sparsentan is a dual-acting, selective antagonist for endothelin type A receptor (ETAR) and angiotensin II receptor type 1 (AT1R). Inhibition of these receptors leads to suppression of proteinuria and glomerulosclerosis during the development and progression of kidney disease.

Qalsody (tofersen) received a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances, for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene.
ALS is a severe neurological disease that leads to paralysis and spasticity. The mean survival time with ALS is two to five years. There is only one authorised treatment for ALS, and the unmet medical need remains high. In a small proportion of ALS patients, the disease is caused by a mutation in the SOD1 gene, which results in the production of defective SOD1 proteins and causes aberrations in neurons.
Qalsody is an antisense oligonucleotide that binds to the mRNA of the SOD1 gene to reduce SOD1 protein production. By reducing the amount of defective SOD1 protein, this medicine is expected to improve the symptoms of ALS.
The EMA has published a press release on Qalsody.

Tizveni (tislelizumab) has received a positive opinion for the following indications:
1.)    Tizveni in combination with pemetrexed and platinum-containing chemotherapy is indicated for the first-line treatment of adult patients with non-squamous non-small cell lung cancer whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have:
• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
• metastatic NSCLC
2.)    Tizveni in combination with carboplatin and either paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous non-small cell lung cancer who have:
• locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
• metastatic NSCLC.
3.)    Tizveni as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.
Non-small cell lung cancer (NSCLC) is a serious and often fatal disease that accounts for 80 to 85% of all lung cancers. A significant number of patients present with driver mutations in oncogenes, some of which can be aimed at by targeted therapies. Patients without known driver mutations need broader-acting therapies.
Tislelizumab is a checkpoint inhibitor, targeting PD-1 (programmed cell death protein 1) on immune cells. Many cancer cells express a PD-1 ligand. Since activation of PD-1 down-regulates the immune system, blocking of the interaction between PD-1 and PD-L1 enhances the T cell responses against the cancer cells. Furthermore, tislelizumab was engineered to reduce Fc-mediated effector protein bindings, thereby reducing the antibody- and complement-dependent cytotoxicity.

Voydeya (danicopan) received a positive opinion as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.
PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.
Danicopan is an orally available inhibitor of the alternative pathway of the complement system. When combined with a complement C5 inhibitor, haemolysis and increased haemoglobin levels can be prevented. The development of Voydeya was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support.
The EMA has published a press release on Voydeya.

Zynyz (retifanlimab) has received a positive opinion for the first-line treatment, as monotherapy, of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) not amenable to curative surgery or radiation therapy.
Merkel cell carcinoma is a rare, aggressive malignancy of the skin with a poor outcome when advanced. The carcinoma originates predominantly on light-exposed parts of the skin. The disease primarily affects old and/or immunosuppressed patients.
Like Tizveni/tislelizumab (see information above), retifanlimab is also a checkpoint inhibitor targeting PD-1.
 

Carvykti (ciltacabtagene autoleucel): extension of indication to include treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor and have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.
Carvykti is already authorised for a later line of treatment of multiple myeloma. 

Cibinqo (abrocitinib): extension of indication to include the treatment of moderate-to-severe atopic dermatitis in adolescents 12 years and older who are candidates for systemic therapy.
Cibinqo is already authorised for adult patients with moderate-to-severe atopic dermatitis.

Kalydeco (ivacaftor): extension of indication to include the treatment, as monotherapy, of infants aged at least 1 month, toddlers and children weighing 3 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
Kalydeco is already authorised for the treatment of patients aged 4 months and above, weighing at least 3 kg, carrying one of the above-listed mutations. In addition, it is authorised in a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of paediatric patients aged 2 to less than 6 years who have at least one F508del mutation in the CFTR gene.

Keytruda (pembrolizumab): extension of indication to include Keytruda, in combination with platinum-containing chemotherapy as neoadjuvant treatment, and then continued as monotherapy as adjuvant treatment, for the treatment of resectable non‑small cell lung carcinoma at high risk of recurrence in adults.
Keytruda is already authorised for the treatment of various types of solid cancer, including NSCLC in different therapeutic settings and lines of therapy. 

Reblozyl (luspatercept): extension of indication to include the treatment of all patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS), regardless of age, prior therapies and eligibility for erythropoietin-based therapy.
Reblozyl is already authorised for adults only, with some restrictions regarding symptoms and prior therapies.

Xromi (hydroxycarbamide): extension of indication to include the prevention of vaso-occlusive complications of Sickle Cell Disease (SCD) in patients over 9 months of age.
Xromi is already authorised for the treatment of SCD in older children from 2 years of age.
 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage: 

Elucirem and Vueway (identical duplicate products, containing gadopiclenol) are for diagnostic use only. They are indicated in adults and children aged 2 years and older for contrast-enhanced magnetic resonance imaging (MRI) to improve detection and, visualization of pathologies with disruption of the blood-brain-barrier (BBB) and/or abnormal vascularity of:
-    the brain, spine, and associated tissues of the central nervous system (CNS);
-    liver, kidney, pancreas, breast, lung, prostate, and musculoskeletal system.
They should be used only when diagnostic information is essential and not available with unenhanced MRI. 
EPAR Elucirem/EPAR Vueway.

Omjjara (momelotinib) is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.
EPAR Omjjara.

Velsipity (etrasimod) is indicated for the treatment of patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.
EPAR Velsipity.

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation
https://www.ema.europa.eu/en/glossary/exceptional-circumstances
https://www.ema.europa.eu/en/news/new-treatment-rare-motor-neurone-disease-recommended-approval
https://www.ema.europa.eu/en/human-regulatory-overview/research-and-development/prime-priority-medicines
https://www.ema.europa.eu/en/news/first-oral-treatment-against-residual-haemolytic-anaemia-patients-paroxysmal-nocturnal-haemoglobinuria
https://www.ema.europa.eu/en/documents/assessment-report/elucirem-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/vueway-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/omjjara-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/velsipity-epar-public-assessment-report_en.pdf

Exblifep (cefepime / enmetazobactam) received a positive opinion for the treatment of the following infections in adults:
• Complicated urinary tract infections (cUTI), including pyelonephritis
• Hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP),
and for the treatment of patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above.
Cefepime and enmetazobactam are two antibiotics. Cefepime is a cephalosporin and directly inhibits bacterial cell wall synthesis, while enmetazobactam inhibits a bacterial enzyme called beta-lactamase. This enzyme is able to hydrolyse cephalosporins. Thus, as a combination treatment, enmetazobactam protects cefepime from being destroyed by beta-lactamase, enabling its anti-bacterial activity.

Ryzneuta (efbemalenograstim alfa) received a positive opinion for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).
Cytotoxic chemotherapy is often accompanied by neutropenia, a decrease in a certain kind of blood cells which play an important role in the immune system. Neutropenia is a severe side effect of chemotherapy, which can lead to an increased risk of infection. The active substance in Ryzneuta is a haematopoietic growth factor which increases the production and differentiation of mature and functionally active neutrophils from bone marrow precursor cells
 

Abecma (idecabtagene vicleucel): extension of indication to include treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least two prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti CD38 antibody and have demonstrated disease progression on the last therapy.
Abecma is already authorised for a later line of treatment of RRMM.

Prevenar 20 / previously Apexxnar (pneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)): extension of indication to include the active immunisation for the prevention of invasive disease, pneumonia, and acute otitis media caused by Streptococcus pneumoniae in infants, children, and adolescents from 6 weeks to less than 18 years of age.
Prevenar 20 / Apexxnar is already authorised for the active immunisation of adults.

Aspaveli (pegcetacoplan): extension of indication to include the treatment, as monotherapy, of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.
Aspaveli is already authorised for the second-line treatment of anaemic PNH patients.

Retsevmo (selpercatinib): extension of indication to include the treatment of adults and adolescents 12 years and older with advanced RET fusion-positive thyroid cancer who are radioactive iodine-refractory (if radioactive iodine is appropriate).
Retsevmo is already authorised for the treatment of advanced RET fusion-positive thyroid cancer in a different second-line setting, and the first-line treatment of medullary thyroid cancer (MTC) and non-small cell lung cancer (NSCLC).

 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage: 

Agamree (vamorolone) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. 
EPAR Agamree.
Elrexfio (elranatamab) is indicated as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. 
EPAR Elrexfio.
Loargys (pegzilarginase) is indicated for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. 
EPAR Loargys.
Rezzayo (rezafungin) is indicated for the treatment of invasive candidiasis in adults.
EPAR Rezzayo.
Veoza (fezolinetant) is indicated for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. 
EPAR Veoza.
Spexotras (trametinib) is indicated for the following indications:
Low-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.
High-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.
EPAR Spexotras.
 

-    Troriluzole - Orphan - is indicated for the treatment of adult patients with spinocerebellar ataxia genotype 3 (SCA3)
-    Mirvetuximab soravtansine - Orphan - treatment of ovarian, fallopian tube, or primary peritoneal cancer
-    Tiratricol - Orphan - treatment of monocarboxylate transporter 8 (MCT8) deficiency
-    Eplontersen - Orphan - indicated for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (ATTRv).
-    Marstacimab - Orphan - is indicated for routine prophylaxis of bleeding episodes in patients with haemophilia A or haemophilia B
-    Elafibranor - Orphan - treatment of primary biliary cholangitis (PBC)
-    Sotatercept - Orphan - Accelerated review - treatment of pulmonary arterial hypertension in adults 
-    Clascoterone - indicated for the topical treatment of acne vulgaris in adults and adolescents

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation
https://www.ema.europa.eu/en/glossary/exceptional-circumstances
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/documents/assessment-report/apretude-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/enrylaze-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/litfulo-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/lyfnua-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/orserdu-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/talvey-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/tepkinly-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/tevimbra-epar-public-assessment-report_en.pdf
 

Casgevy (exagamglogene autotemcel) received a positive opinion, recommending the granting of a conditional marketing authorisation (CMA), for the treatment of transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD). The full indication is:
β-thalassemia
Casgevy is indicated for the treatment of transfusion-dependent β-thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.
Sickle cell disease
Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.
β-thalassemia and sickle cell disease are two inherited disorders of the red blood cells. Different mutations lead to impaired production of haemoglobin and thus, to problems in red blood cell function. Casgevy is a cellular therapy, consisting of the patient’s own haematopoietic stem cells which are genetically modified to foster normal gene expression in red blood cells. Casgevy needs to be administered only once.

Skyclarys (omaveloxolone) received a positive opinion for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
Friedreich’s ataxia is a hereditary progressive degenerative disorder and is caused by mutations in a gene encoding a mitochondrial protein, frataxin. The impaired production of this protein results in progressive neurological symptoms like movement and speech disorders, and sensory loss. Skyclarys activates a protein involved in the cellular response to oxidative stress, which is involved in the pathophysiological mechanism of the disease.

Velsipity (etrasimod) received a positive opinion for the treatment of patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.
UC is a chronic inflammatory disease that affects the colon. Although the aetiology is not fully understood, many factors seem to be involved in the pathogenesis of the disease, including altered immune responses, environmental and genetic factors. Velsipity is a selective immunosuppressant. It blocks receptors expressed on lymphocytes and thus reduces lymphocyte migration into sites of inflammation.

Arpraziquantel (arpraziquantel) received a positive opinion for the treatment of schistosomiasis in young children aged 3 months to 6 years. The application for marketing authorisation was submitted under the EU-M4all procedure for high-priority medicines for human use intended for markets outside the EU.
Schistosomiasis is a parasitic disease caused by trematode worms, and causes damage of several organs, as the bladder, the kidneys and the liver. The parasite infection occurs in tropical and subtropical regions and mainly affects people without access to clean water. Arpraziquantel is a derivative of praziquantel, the standard treatment of Schistosomiasis since decades. Arpraziquantel has been developed to provide an age-appropriate formulation for young children, as it can be administered as a dispersible tablet.

HyQvia (human normal immunoglobulin): extension of indication to include HyqVia as an immunomodulatory therapy in adults, children and adolescents (0 to 18 years) in:
-    Chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with IVIg.
Hyqvia is already authorised as a replacement therapy in patients with primary and secondary immunodeficiency syndromes.

Metalyse (tenectaplase): extension of indication to include the thrombolytic treatment of acute ischaemic stroke (AIS) within 4.5 hours from last known well and after exclusion of intracranial haemorrhage.
Metalyse is already authorised, with a different strength, for the thrombolytic treatment of suspected myocardial infarction.

VeraSeal (human fibrinogen / human thrombin): extension of indication to include the supportive treatment in children of all age groups where standard surgical techniques are insufficient:
- for improvement of haemostasis.
- as suture support: in vascular surgery.
VeraSeal is already authorised for the treatment of adult patients in the same indications.

Zinplava (bezlotoxumab): extension of indication to include the preventive treatment of paediatric patients 1 year of age and older at high risk for recurrence of Clostridioides difficile infection (CDI).
Zinplava is already authorised for the treatment of adult patients in the same indication.

Fexinidazole Winthrop (fexinidazole): extension of indication for the treatment of both first-stage (haemo-lymphatic) and second-stage (meningo-encephalitic) of human African trypanosomiasis (HAT) due to Trypanosoma brucei rhodesiense (r-HAT) in adults and children ≥ 6 years old and weighing ≥ 20 kg.
Fexinidazole Winthrop is already authorised for the treatment of HAT due to Trypanosoma brucei gambiense (g‑HAT).

 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage: 

Jaypirca (pirtobrutinib) is indicated for the treatment, as monotherapy, of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton’s tyrosine kinase (BTK) inhibitor. 

EPAR Jaypirca.

Inaqovi (decitabine / cedazuridine) is indicated for the treatment, as monotherapy, of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy. 

EPAR Inaqovi.

Aqumeldi (enalapril maleate) is indicated for the treatment of heart failure in children from birth to less than 18 years.

EPAR Aqumeldi.

Ebglyss (lebrikizumab) is indicated for the treatment of moderate-to-severe atopic dermatitis in adults and adolescents 12 years and older with a body weight of at least 40 kg who are candidates for systemic therapy.

EPAR Ebglyss.

Finlee (dabrafenib) has the following indications:
Low-grade glioma
Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.
High-grade glioma
Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

EPAR Finlee.

Vanflyta (quizartinib) is indicated in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by Vanflyta single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD (Feline McDonough Sarcoma-like tyrosine kinase 3 internal tandem duplication) positive.

EPAR Vanflyta.

Yorvipath (palopegteriparatide) is a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.

EPAR Yorvipath.

Zilbrysq (zilucoplan) is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

EPAR Zilbrysq.

 

-    aztreonam / avibactam – treatment of infections (cIAI, HAP, VAP, and cUTI), and aerobic Gram-negative infections with limited treatment options
-    In vitro diagnostic medical device – next generation sequencing (NGS) assay for tumour mutation profiling
-    erdafitinib – treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC)
-    amino acids – Orphan – treatment of decompensation episodes in maple syrup urine disease (MSUD) patients
-    imetelstat – Orphan – treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation
https://www.ema.europa.eu/en/partners-networks/international-activities/medicines-assessed-under-eu-m4all-procedure 
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/en/documents/assessment-report/jaypirca-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/inaqovi-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/aqumeldi-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/ebglyss-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/finlee-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/vanflyta-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/yorvipath-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/en/documents/assessment-report/zilbrysq-epar-public-assessment-report_en.pdf
 

Krazati (adagrasib) as monotherapy has received a positive opinion for a conditional marketing authorisation (CMA) for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and disease progression after at least one prior systemic therapy.
NSCLC is a serious and often fatal disease that accounts for over 85% of all lung cancers. KRAS-G12C is a driver mutation in many cancers and it represents about 13 % of all mutations in NSCLC. KRAS is a GTPase encoded by a proto-oncogene and the mutated KRAS-G12C is a constitutively active form of the enzyme. Krazati binds KRAS-G12C and prevents downstream signalling by locking the mutated protein in an inactive state.

Omjjara (momelotinib) received a positive opinion for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.
Myelofibrosis is an uncommon type of myeloproliferative neoplasm, in which fibrous tissue in the bone marrow interferes with normal blood cell production. This results in extramedullary movement to other organs, such as the spleen and liver, and results in enlargement of these organs. Symptoms of the disease include bone pain, tiredness, weakness, weight loss, fever and bleeding.
Primary myelofibrosis occurs de novo, while secondary forms of myelofibrosis arise from the myeloproliferative diseases essential thrombocythemia or polycythemia vera.
Myelofibrosis is associated with an aberrant expression of the tyrosine kinase JAK, which is involved in cytokine signalling pathways. This leads to the increased production of immature blood cells. Omjjara is an inhibitor of JAK 1 and 2.

Rystiggo (rozanolixizumab) received a positive opinion as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
gMG is a rare autoimmune disorder that causes muscle weakness. Around 90% of patients have detectable levels of autoantibodies in serum and the most common target AChR at the neuromuscular junction (NMJ), leading to failure of neuromuscular transmission. Rystiggo is a monoclonal antibody which targets the neonatal Fc receptor (FcRn). Thus, the binding of IgG antibodies to FcRn is suppressed and the intracellular degradation of the pathogenic autoantibodies is stimulated. 

Spexotras (trametinib) has received a positive opinion for the following indications:
Low-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.
High-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.
Gliomas are a group of primary tumours of the central nervous system with glial origin. LGG are rare and HGG even rarer tumours in the paediatric population. The BRAF V600E mutation is present in around 17% of paediatric LGG cases and in 6% of paediatric and young adult cases of HGG. Spexotras is a small molecule allosteric inhibitor of the mitogen-activated protein kinase, MEK, which is an important part of a signalling pathway regulating proliferation, differentiation and apoptosis in cancer cells. Dabrafenib is a small molecule inhibitor of the V600-mutant BRAF kinase. 

Ayvakyt (avapritinib): extension of indication to include the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment.
Ayvakyt is already authorised for the treatment of gastrointestinal stromal tumours (GIST), aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) and mast cell leukaemia (MCL).

Evkeeza (evinacumab): extension of indication, as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies, for the treatment paediatric patients aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH).
Evkeeza is already authorised for the treatment of adults and adolescents from 12 years of age.

Fluad tetra (influenza vaccine (surface antigen, inactivated, adjuvanted)): extension of indication to include prophylaxis of influenza in adults 50 years of age and older.
Fluad tetra is already authorised for adults from 65 years of age.

Jardiance (empagliflozin): extension of indication to include children aged 10 years and above for the treatment of insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise,
• as monotherapy when metformin is considered inappropriate due to intolerance
• in addition to other medicinal products for the treatment of diabetes
Jardiance is already authorised for the treatment of insufficiently controlled diabetes mellitus in adult patients. Moreover, it is authorised for the treatment of symptomatic chronic heart failure and chronic kidney disease in adults.

Keytruda (pemprolizumab): extension of indication to include Keytruda, in combination with gemcitabine-based chemotherapy, for the first-line treatment of locally advanced unresectable or metastatic biliary tract carcinoma in adults.
Keytruda is already authorised for the treatment of various types of solid cancer. In September and October 2023, Keytruda received positive opinions for the adjuvant treatment of adults with non-small cell lung carcinoma who are at high risk of recurrence following complete resection and platinum-based chemotherapy, as well as for the first-line treatment, in combination with fluoropyrimidine and platinum-containing chemotherapy, of locally advanced unresectable or metastatic HER2-negative gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1. Both EC decisions are pending. 

Mounjaro (tirzepatide): extension of indication to include, as an adjunct to a reduced-calorie diet and increased physical activity for weight management, including weight loss and weight maintenance, in adults with an initial Body Mass Index (BMI) of
• ≥ 30 kg/m2 (obesity) or
• ≥ 27 kg/m2 to < 30 kg/m2 (overweight) in the presence of at least one weight-related comorbid condition (e.g., hypertension, dyslipidaemia, obstructive sleep apnoea, cardiovascular disease, prediabetes, or type 2 diabetes mellitus).
Mounjaro is already authorised for the treatment of adults with insufficiently controlled type 2 diabetes mellitus.

Nexobrid (concentrate of proteolytic enzymes enriched in bromelain): extension of indication to include all age groups for removal of eschar in patients with deep partial- and full-thickness thermal burns.
Nexobrid is already authorised for the treatment of adult patients with the same indication.

Talzenna (talazoparib): extension of indication to include, in combination with enzalutamide, the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated.
Talzenna is already authorised for the treatment of breast cancer.

Veltassa (patiromer): extension of indication to include the treatment of hyperkalaemia in adolescents aged 12 to 17 years.
Veltassa is already authorised for the treatment of adult patients with the same indication.

 

Links, in order or appearance:
https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation

Agamree (vamorolone) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.
DMD is a rare, severe muscle wasting disease, which affects boys from birth and is invariably fatal due to the lack of dystrophin, a structural and regulatory protein at the membranes of muscle fibres. It is caused by errors in the gene encoding dystrophin, an essential cohesive protein which connects muscle fibers with the surrounding extracellular matrix. The dystrophin gene is located on the X-chromosome, therefore DMD is inherited in an X-chromosomal recessive manner. Vamorolone is a corticosteroid that was designed to separate sub-activities of traditional glucocorticoids (such as prednisone). One important characteristic is the stabilisation of plasma membranes with protection against membrane injury.

Elrexfio (elranatamab) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed and refractory multiple myeloma (MM), who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
MM is a type of blood cancer characterised by the malignant proliferation of plasma cells that consistently express the B-cell maturation antigen (BCMA). BCMA is not expressed in normal non-haematopoietic cells. Elrexfio is a bispecific antibody that targets CD3, present on the surface of T-cells, and BCMA. Elrexfio binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with BCMA, T-cell activation occurs, resulting in T-cell-mediated B-cell lysis.

Elucirem/Vueway (gadopiclenol) is for diagnostic use only. It is indicated in adults and children aged 2 years and older for contrast-enhanced magnetic resonance imaging (MRI) to improve detection and, visualization of pathologies with disruption of the blood-brain-barrier (BBB) and/or abnormal vascularity of:
-    the brain, spine, and associated tissues of the central nervous system (CNS);
-    liver, kidney, pancreas, breast, lung, prostate, and musculoskeletal system.
It should be used only when diagnostic information is essential and not available with unenhanced MRI.
Gadopiclenol is a macrocyclic contrast agent based on gadolinium. Compared with standard gadolinium-based contrast agents, gadopiclenol shows differing spectroscopic characteristics and can thus be used in lower doses.

Loargys (pegzilarginase) received a positive opinion for a marketing authorisation under exceptional circumstances for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.
Hyperargininaemia is a rare, debilitating, progressive, inherited, metabolic disease. It is characterised by deficiency of the arginase 1 enzyme and associated with the persistent elevation of plasma arginine leading to progressive neurodegenerative symptoms. Patients present with the development of progressive spasticity with greater severity in the lower limbs. They may also develop seizures and gradually lose intellectual attainments. Treatment with Loargys aims at substitution of the deficient ARG1 enzyme activity.

Rezzayo (rezafungin) is indicated for the treatment of invasive candidiasis in adults.
Invasive candidiasis is a serious and life-threatening infection caused by Candida species, associated with high mortality rates. It can cause invasive disease characterised by both widely disseminated and/or bloodstream infections. Rezafungin selectively inhibits 1,3-β-D-glucan synthase, an enzyme present in fungal, but not mammalian, cells. This results in inhibition of the formation of 1,3-β-D-glucan, an essential component of the fungal cell wall.

Veoza (fezolinetant) is indicated for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause.
VMS, also referred to as hot flashes, involve sudden episodes of heat in the upper body, particularly in the head, neck, chest and upper back. Fezolinetant inhibits the neurokinin 3 receptor which is expressed in the central nervous system. This inhibition results in a blockade of a signaling pathway involved in the thermoregulatory center of the brain, and ultimately leads to reduced VMS.

 

Brukinsa (zanubrutinib): extension of indication to, in combination with obinutuzumab, the treatment of adult patients with refractory or relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies.
Brukinsa was already authorised for the treatment of Waldenström’s macroglobulinaemia, marginal zone lymphoma and chronic lymphocytic leukaemia.

Imfinzi (durvalumab): extension of indication to include, as monotherapy, the first line treatment of adults with advanced or unresectable hepatocellular carcinoma (HCC).
Imfinzi was already authorised, as a combination therapy with chemotherapeutic agents and/or tremelimumab, for the treatment of HCC, small cell lung cancer, biliary tract cancer, and non-small cell lung cancer.

Jemperli (dostarlimab): extension of indication to include, in combination with carboplatin and paclitaxel, the treatment of adult patients with mismatch repair deficient (dMMR)/ microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy.
Jemperli was already authorised for the second-line treatment of the same subtype of endometrial cancer.

Keytruda (pembrolizumab): extension of indication to include, in combination with fluoropyrimidine and platinum-containing chemotherapy, the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1.
Keytruda was already authorised for the treatment of various types of cancer, including HER2-positive gastric or gastro-oesophageal junction (GEJ) adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1. Last month, Keytruda received a positive CHMP opinion for the adjuvant treatment of adults with non-small cell lung carcinoma who are at high risk of recurrence following complete resection and platinum-based chemotherapy. European commission (EC) decision is pending.

Praluent (alirocumab): extension of indication to the treatment of paediatric patients 8 years of age and older with heterozygous familial hypercholesterolaemia (HeFH) as an adjunct to diet, in combination with a statin, or statin with other lipid lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin or, alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.
Praluent was already authorised for the treatment of adults with primary hypercholesterolaemia (heterozygous familial and non-familial), mixed dyslipidaemia, or with established atherosclerotic cardiovascular disease.

Prevymis (letermovir): extension of indication to include prophylaxis of CMV disease in CMV-seronegative adults who have received a kidney transplant from a CMV-seropositive donor [D+/R-].
Prevymis was already authorised for the prophylaxis of CMV reactivation and disease in CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant.

Rubraca (rucaparib): extension of indication to include, as monotherapy, the maintenance treatment of adult patients with advanced (FIGO Stages III and IV) high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy.
Rubraca was already authorised for the maintenance treatment of the same cancer types in patients who are in response to an ongoing platinum-based chemotherapy.  

Veyvondi (vonicog alfa): extension of indication to include the prevention of haemorrhage or surgical bleeding in adults (age 18 years and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.
Veyvondi was already authorised for the treatment of haemorrhage or surgical bleeding in the same patient population.

 

Newly published EPARs:
The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Apretude (capotegravir) is indicated in combination with safer sex practices for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 infection in high-risk adults and adolescents, weighing at least 35 kg. EPAR Apretude.

Enrylaze (crisantaspase) is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL) in adult and paediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. EPAR Enrylaze.

Litfulo (ritlecitinib tosilate) is indicated for the treatment of severe alopecia areata in adults and adolescents 12 years of age and older. EPAR Litfulo.

Lyfnua (gefapixant) is indicated in adults for the treatment of refractory or unexplained chronic cough. EPAR Lyfnua.

Orserdu (elacestrant) monotherapy is indicated for the treatment of postmenopausal women, and men, with estrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor. EPAR Orserdu.

Talvey (talquetamab) is indicated as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. EPAR Talvey.

Tepkinly (epcoritamab) as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. EPAR Tepkinly.

Tevimbra (tislelizumab) as monotherapy is indicated for the treatment of adult patients with unresectable, locally advanced or metastatic oesophageal squamous cell carcinoma after prior platinum-based chemotherapy. EPAR Tevimbra.

 

-    Avacincaptad pegol - is indicated for the treatment of adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
-    Crovalimab - treatment of paroxysmal nocturnal haemoglobinuria
-    Dasiglucagon - treatment of severe hypoglycemia in patients with diabetes
-    Delgocitinib -  treatment of moderate to severe chronic hand eczema (CHE)
-    Donanemab - to slow disease progression in adult patients with Alzheimer’s disease (AD).
-    Fruquintinib – Treatment of metastatic colorectal cancer. Fruquintinib is an orally available inhibitor of vascular endothelial growth factor receptors (VEGFRs) 1, 2, and 3.
-    Givinostat - treatment of Duchenne muscular dystrophy (DMD)
-    In vitro diagnostic medical device - detection of HER2 antigen
-    In vitro diagnostic medical device - detection of PD-L1 protein.
-    In vitro diagnostic medical device - immunohistochemical assay utilising an anti-PDL1 monoclonal primary antibody
-    In vitro diagnostic medical device - in vitro diagnostic device for laboratory use, intended for the qualitative detection of BRAF V600 mutations in DNA extracted from formalinfixed, paraffin-embedded human tissue.
-    Liquid ethanolic extract 30 per cent (w/w) of allium cepa fresh bulb and citrus limon fresh fruit / dry aqueous extract of paullinia cupana seed / dry hydroethanolic extract of theobroma cacao seed - treatment of alopecia areata in children and adolescents
-    Lutetium (177Lu) chloride - radiolabelling of carrier molecules, which have been specifically developed for radiolabelling with this radionuclide.
-    Macitentan / tadalafil - treatment of pulmonary arterial hypertension (PAH) in adult patients
-    Meningococcal group a, b, c, w and y vaccine - indicated for active immunisation to prevent invasive disease caused by Neisseria meningitidis groups A, B, C, W, and Y
-    Odronextamab – Orphan – Treatment of blood cancers (follicular lymphoma (FL) or diffuse large B cell lymphoma (DLBCL) and large cell lymphoma).
-    Single-stranded 5' capped mRNA encoding the respiratory syncytial virus glycoprotein f stabilized in the prefusion conformation – OPEN - Prevention of lower respiratory tract disease (LRTD) and acute respiratory disease (ARD) caused by respiratory syncytial virus (RSV)
-    Vilobelimab - treatment of adult patients with SARS-CoV-2 induced septic acute respiratory distress syndrome (ARDS) receiving invasive mechanical ventilation (IMV) or extracorporeal membrane oxygenation (ECMO).
-    Zapomeran - active immunisation to prevent COVID-19
-    Zolbetuximab – Orphan – Treatment of locally advanced unresectable or metastatic HER2 negative gastric or gastro-oesophageal junction (GEJ) adenocarcinoma.
-    Zoonotic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted) - active immunisation against H5 subtype of Influenza A virus

 

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp
https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation
https://www.ema.europa.eu/en/glossary/exceptional-circumstances
https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/documents/assessment-report/apretude-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/enrylaze-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/litfulo-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/lyfnua-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/orserdu-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/talvey-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/tepkinly-epar-public-assessment-report_en.pdf
https://www.ema.europa.eu/documents/assessment-report/tevimbra-epar-public-assessment-report_en.pdf

 

Aqumeldi (enalapril maleate) is indicated for the treatment of heart failure in children from birth to less than 18 years. Heart failure is an impairment of the blood pumping function of the heart and reduced efficiency of the heart muscle. In children it is most commonly caused by congenital heart defects. The renin-angiotensin system (RAS) is the endocrine system involved in the regulation of blood pressure and vascular resistance, amongst other. The angiotensin-converting enzyme (ACE), a part of RAS, converts angiotensin I into angiotensin II, an active vasoconstrictor, thereby increasing blood pressure. Aqumeldi is an ACE inhibitor, a class of drugs also indicated for the treatment of blood pressure. Aqumeldi is a hybrid medicine of Renitec, which has been authorised in the EU since 1985. Although both contain the same active substance, Aqumeldi is available at lower strength and in a formulation more appropriate for children. The application for Aqumeldi was submitted as a paediatric-use marketing authorisation (PUMA).

Ebglyss (lebrikizumab) is indicated for the treatment of moderate-to-severe atopic dermatitis (AD) in adults and adolescents 12 years and older with a body weight of at least 40 kg who are candidates for systemic therapy. AD is a chronic and complex disease, characterised by dry and pruritic skin. The main contributors seem to be an altered permeability of the skin due to perturbations in the epidermal barrier and an inappropriate immune response, which results in skin inflammation and contributes to the epidermal barrier disruption. Interleukin 13 (IL-13) is believed to be key in the pathophysiology of AD. Ebglyss is a monoclonal antibody that binds and inhibits IL-13, thereby disrupting the cytokine signalling cascade involved in AD.

Finlee (dabrafenib) has received a positive opinion for the following indications:

Low-grade glioma

Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.

High-grade glioma

Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

Gliomas are a group of primary tumours of the central nervous system with glial origin. LGG are rare and HGG even rarer tumours in the paediatric population. The BRAF V600E mutation is present in around 17% of paediatric LGG cases and in 6% of paediatric and young adult cases of HGG. Finlee is a small molecule inhibitor of the V600-mutant BRAF kinase and trametinib is a small molecule inhibitor of the mitogen-activated protein kinase (MEK). Finlee has been developed with a dispersible tablet formulation, suitable for the paediatric population. An application for a pharmaceutical form suitable for the paediatric population, powder for oral solution, of trametinib is currently being assessed by CHMP for in indication in combination with Finlee.

Vanflyta (quizartinib) is indicated in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by Vanflyta single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD positive.

AML is a cancer of the white blood cells characterised by the overproduction of immature myeloid cells. Mutations in FLT3 are one of the most common in newly diagnosed AML, and the internal tandem duplication (ITD) in FLT3 is associated with aggressive malignancies and unfavourable prognosis, due to a high rate of relapse. Vanflyta is a small molecule inhibitor of FLT3, which binds at the ATP-binding pocket, thereby preventing downstream signalling and FLT3-ITD-dependent proliferation.

Yorvipath (palopegteriparatide) is a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism (HPT). HPT is a rare disease characterised by a reduced function of the parathyroid glands, which results in a lower production of PTH. Although HPT can be a result of genetic and autoimmune factors, the majority of the cases arise after complications from neck surgery. The main symptoms of HPT are a consequence of low calcium levels. Yorvipath is a prodrug consisting of the first 34 aminoacids of the endogenous PTH, conjugated to an inert methoxypolyethylene glycol (mPEG) carrier via a linker, which is cleaved after administration releasing the active moiety.

Zilbrysq (zilucoplan) is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. MG is a rare autoimmune disorder that causes muscle weakness. Around 90% of patients have detectable levels of autoantibodies in serum and the most common target AChR at the neuromuscular junction (NMJ), leading to failure of neuromuscular transmission. Activation of the complement system has been shown in the NMJ. Zilbrysq is a macrocyclic peptide that inhibits the protein complement component 5 (C5), a component of the terminal complement system.

Adcetris (brentuximab vedotin): extension of indication to the treatment of adult patients with previously untreated CD30+ Stage III Hodgkin lymphoma in combination with doxorubicin, vinblastine and dacarbazine. Adcetris was already authorised for different stages of Hodgkin lymphoma, and various lines of therapy; for systemic anaplastic large cell lymphoma and cutaneous T-cell lymphoma.

Enhertu (trastuzumab deruxtecan): extension of indication to include, as monotherapy, treatment of adult patients with advanced non-small cell lung cancer whose tumours have an activating HER2 (ERBB2) mutation and who require systemic therapy following platinum-based chemotherapy with or without immunotherapy. Enhertu was already authorised for breast cancer and gastric cancer.

Kaftrio (ivacaftor/tezacaftor/elexacaftor) granules: extension of indication, in a combination regimen with ivacaftor, to include treatment of cystic fibrosis in patients aged 2 to less than 6 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kaftrio was already authorised, as capsules, in adults and children from 6 years of age.

Kalydeco (ivacaftor) granules: extension of indication, in a combination regimen with ivacaftor/tezacaftor/elexacaftor, to include treatment of cystic fibrosis in patients aged 2 to less than 6 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kalydeco was already authorised, as tablets, in adults and children from 6 years of age with at least one F508del mutation in the CFTR gene, and as monotherapy in children from 4 months of age with different mutations.

Keytruda (pembrolizumab): extension of indication to include, as monotherapy, the adjuvant treatment of adults with non-small cell lung carcinoma who are at high risk of recurrence following complete resection and platinum-based chemotherapy. Keytruda was already authorised for the treatment of melanoma, non-small cell lung carcinoma, classical Hodgkin lymphoma, urothelial carcinoma, head and neck squamous cell carcinoma, renal cell carcinoma, colorectal cancer, oesophageal carcinoma, breast cancer, endometrial carcinoma, gastric or gastro-oesophageal junction (GEJ) adenocarcinoma, cervical cancer and several microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) cancers.

Nordimet (methtotrexate): update of indication to the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy severe and severe psoriatic arthritis in adult patients. Nordimet was previously authorised only in severe recalcitrant disabling psoriasis, not adequately responsive to other forms of therapy such as phototherapy, psoralens and ultraviolet A (PUVA), and retinoids, and severe psoriatic arthritis in adult patients. Nordimet was already authorised for the treatment of active rheumatoid arthritis, juvenile idiopathic arthritis and Crohn's disease.

Olumiant (baricitinib): extension of indication to the treatment of moderate to severe atopic dermatitis in paediatric patients 2 years and older who are candidates for systemic therapy. Olumiant was previously only authorised in adults in this indication. Furthermore, Olumiant was already authorised for the treatment of rheumatoid arthritis, alopecia areata and juvenile idiopathic arthritis.

Pepaxti (melphalan flufenamide) extension of indication, in combination with dexamethasone, to the treatment of adult patients with multiple myeloma who have received at least two prior lines of therapies, whose disease is refractory to lenalidomide and the last line of therapy. Pepaxti was previously only authorised in patients who had received at least 3 lines of therapy and were refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody.

Ryeqo (relugolix / estradiol / norethisterone acetate): extension of indication to the symptomatic treatment of endometriosis in women with a history of previous medical or surgical treatment for their endometriosis. Ryeqo was previously authorised for the symptomatic treatment of uterine fibroids.

Takhzyro (lanadelumab): extension of indication to the routine prevention of recurrent attacks of hereditary angioedema in patients aged 2 years and older. Takhzyro was previously authorised in patients 12 years of age and older.

Voxzogo (vosoritide): extension of indication to the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. Voxzogo was previously authorised in patients 2 years of age and older.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Abrysvo (Respiratory syncytial virus vaccines) is indicated for:

Passive protection against lower respiratory tract disease caused by respiratory syncytial virus (RSV) in infants from birth through 6 months of age following maternal immunisation during pregnancy.

Active immunisation of individuals 60 years of age and older for the prevention of lower respiratory tract disease caused by RSV.

EPAR Abrysvo.

Aquipta (atogepant) is indicated for prophylaxis of migraine in adults who have at least 4 migraine days per month. EPAR Aquipta.

Camzyos (mavacamten) is indicated for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy in adult patients. EPAR Camzyos.

Pylclari (piflufolastat [18F]) is indicated for the detection of prostate-specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer (PCa) in the following clinical settings:

Primary staging of patients with high-risk PCa prior to initial curative therapy,

To localize recurrence of PCa in patients with a suspected recurrence based on increasing serum prostate-specific antigen (PSA) levels after primary treatment with curative intent.

EPAR Pylclari.

Ztalmy (ganaxolone) is indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older. EPAR Ztalmy.

Apadamtase alfa - Orphan - Treatment of congenital thrombotic thrombocytopenic purpura due to ADAMTS13 deficiency.

Capivasertib - Is indicated in combination with fulvestrant for the treatment of adult patients with hormone receptor positive, human epidermal growth factor receptor 2 (HER2) negative (defined as IHC 0 or 1+, or IHC 2+/ISH-) locally advanced or metastatic breast cancer following recurrence or progression on or after an endocrine based regimen.

Efanesoctocog alfa - Orphan - Treatment and prophylaxis of bleeding in patients with haemophilia A.

Fidanacogene elaparvovec - Orphan - Advanced therapy medicinal product - Indicated for the treatment of severe and moderately severe haemophilia B.

Insulin icodec - Treatment of diabetes mellitus in adults.

Iptacopan - Orphan - Treatment of paroxysmal nocturnal haemoglobinuria.

Vibegron - Treatment of micturition frequency and/or urgency incontinence as may occur in adult patients with Over Active Bladder syndrome.

Links, in order or appearance:

Committee for Medicinal Products for Human Use (CHMP) (EMA)

Conditional marketing authorisation (EMA)

European public assessment reports: background and context (EMA)

Assessment report: Abrysvo (pdf)

Assessment report: Aquipta (pdf)

Assessment report: Camzyos (pdf)

Assessment report: Pylclari (pdf)

Assessment report: Ztalmy (pdf)

Abrysvo (Respiratory syncytial virus vaccine) received a positive opinion for the:

• Passive protection against lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in infants from birth through 6 months of age following maternal immunisation during pregnancy;
• Active immunisation of individuals 60 years of age and older for the prevention of LRTD caused by RSV.

RSV is a single-stranded RNA virus that causes the majority of cases of respiratory hospitalization in infants. In adults over 65 years of age, approximately 15% of acute respiratory infections due to RSV lead to hospitalisation. LRTD is characterised by bronchiolitis and pneumonia and it is potentially life-threatening. Abrysvo is a protein-based vaccine that consists of F glycoprotein ectodomains of RSV A and B, expressed in genetically engineered Chinese Hamster Ovary cell lines. There is a particularity of Abrysvo: at variance with other vaccines, the goal of the immunization of pregnant women is the protection not of the mother but of the child who will transplacentally receive the antibodies the mother’s immune system produces against RSV.

Apretude (cabotegravir) received a positive opinion in combination with safer sex practices for pre-exposure prophylaxis (PrEP), to reduce the risk of sexually acquired HIV-1 infection in high-risk adults and adolescents weighing at least 35 kg.

HIV-1 is an RNA retrovirus that causes infection in humans, leading to a progressive failure of the immune system if the infection progresses. Immunocompromised patients are susceptible of contracting life-threatening infections and of developing cancer. By binding to the integrase active site, Apretude blocks the strand transfer step of DNA integration, key to the viral replication cycle.

Enrylaze (crisantaspase) received a positive opinion for the treatment, as a component of a multi-agent chemotherapeutic regimen, of acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL) in adult and paediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to Escherichia coli-derived asparaginase.

LBL, an aggressive non-Hodgkin lymphoma, and ALL are blood cancers characterised by production of large amounts of immature lymphocytes by the blood marrow. ALL and LBL cancer cells are unable to synthesize asparagine, since they lack the necessary enzyme and, therefore, require exogenous sources of asparagine for their survival. Enrylaze is a recombinant form of the enzyme asparaginase, which catalyzes the conversion of L-asparagine into L-aspartic acid. Immunological reactions against previously approved products with asparaginase produced in E. coli may make these unsuitable for some patients. Enrylaze is a recombinant form of the asparaginase produced in Pseudomonas fluorescens and thus allows the treatment of patients who cannot be treated with E. coli-derived asparaginase.

Inaqovi (decitabine / cedazuridine) received a positive opinion for the treatment, as monotherapy, of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy.

AML is a cancer of the white blood cells characterised by the overproduction of immature myeloid cells.

Inaqovi is a fixed-dose combination of the known active substance decitabine and the new active substance cedazuridine intended for oral administration. Decitabine is an antimetabolite (cytidine analogue), authorised in the EU since 2012 as Dacogen for the treatment of adult patients with newly diagnosed de novo or secondary AML, according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy. Dacogen is intended for intravenous administration.

Cedazuridine is a novel cytidine deaminase inhibitor that reduces the hepatic first-pass degradation of decitabine. The enhanced oral availability allows the treatment of patients with tablets (containing both decitabine and cedazuridine) instead of repeated decitabine infusions.

Litfulo (ritlecitinib) received a positive opinion for the treatment of severe alopecia areata (AA) in adults and adolescents 12 years of age and older. AA is an autoimmune disease that causes hair loss in some or all areas of the body, with an average onset between 25 and 35 years of age. Although the pathophysiology of AA is complex and not fully understood, the initiation of the disease seems to be driven by cytotoxic T-cells. This cascade can be blocked by inhibiting enzymes like the Janus kinase/signal transducers and activators of transcription (JAK/STAT) pathway. Litfulo is a selective irreversible inhibitor of enzymes JAK3 and tyrosine-protein kinase expressed in hepatocellular carcinoma (TEC).

Lyfnua (gefapixant) received a positive opinion for the treatment of refractory or unexplained chronic cough (UCC) in adults. Cough is a protective reflex to prevent aspiration into the lung and clear the throat and it has recently been considered as a clinical entity and not only as a symptom. Although the epidemiology of UCC is not well understood, the estimated prevalence is 4 to 11%. Lyfnua is a selective reversible P2X3 receptor antagonist, although it also has activity against the P2X2/3 receptor subtype. These receptors are ATP-gated ion channels and are involved in several physiological and pathological functions including cough, since ATP released in airway tissue triggers certain protective reflex responses via its interaction with these receptors.

Orserdu (elacestrant) received a positive opinion for the treatment, as monotherapy, of postmenopausal women, and men, with estrogen receptor (ER)-positive, human epidermal growth factor receptor (HER) 2-negative, locally advanced or metastatic breast cancer with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor.

Breast cancer is the leading cause of cancer and cancer deaths in women worldwide. Mutation in the ESR1 (estrogen receptor 1) gene are common acquired mutations that confer resistance to endocrine therapy. Orserdu is the first orally available, selective antioestrogen, an antagonist of the ER, or selective ER degrader (SERD), active also against mutated ERs.

Talvey (talquetamab) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed and refractory multiple myeloma (MM), who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

MM is a type of blood cancer characterized by the malignant proliferation of plasma cells. Talvey is a bispecific antibody that targets CD3, present on the surface of T-cells, and GPRC5D, a G protein–coupled receptor of unknown function but highly expressed in multiple myeloma cells. Talvey binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with GPRC5D, T-cell activation occurs, resulting in T-cell-mediated B-cell lysis. Talvey is the first therapy for the treatment of cancer targeting GPRC5D.

Tepkinly (epcoritamab) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

DLBCL is an aggressive cancer of the B-lymphocytes and it is the most common form of non-Hodgkin lymphoma. As many mature B-cell lymphomas, DLBCL express the surface antigen CD20. Tepkinly is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Tepkinly binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated B-cell lysis.

Tevimbra (tislelizumab) received a positive opinion for the treatment, as monotherapy, of adult patients with unresectable, locally advanced or metastatic oesophageal squamous cell carcinoma after prior platinum-based chemotherapy.

Although oesophageal cancer is a rare disease in Europe, it is one of the most common cancers worldwide and it remains highly fatal.

Tevimbra is a checkpoint inhibitor, targeting PD-1 (programmed cell death protein 1) on immune cells. Many cancer cells express a PD-1 ligand. Since activation of PD-1 down-regulates the immune system, blocking of the interaction between PD-1 and PD-L1 enhances the T cell responses against the cancer cells. Furthermore, Tevimbra was engineered to reduce Fc-mediated effector protein bindings, thereby reducing the antibody- and complement-dependent cytotoxicity.

Bylvay (odevixibat): extension of indication to the treatment of cholestatic pruritus in Alagille syndrome in patients aged 6 months or older. Bylvay was already authorised for the treatment of progressive familial intrahepatic cholestasis.

Olumiant (baricitinib): extension of indication to the treatment of active juvenile idiopathic arthritis in patients 2 years of age and older who have had an inadequate response or intolerance to one or more prior conventional synthetic or biologic disease-modifying antirheumatic drugs (DMARDs):

• Polyarticular juvenile idiopathic arthritis (polyarticular rheumatoid factor positive or negative, extended oligoarticular),
• Enthesitis related arthritis, and
• Juvenile psoriatic arthritis.

Baricitinib may be used as monotherapy or in combination with methotrexate. Olumiant was already authorised for the treatment rheumatoid arthritis, atopic dermatitis and areata.

Ervebo (Ebola Zaire Vaccine [rVSV∆G-ZEBOV-GP, live]): extension of indication to the active immunisation of individuals 1 year of age or older to protect against Ebola Virus Disease caused by Zaire Ebola virus. Ervebo was already authorised in patients 18 years of age and older.

Evrysdi (risdiplam): extension of indication to the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi was already authorised in patients 2 months of age and older.

Foclivia (pandemic influenza vaccine [H5N1] [surface antigen, inactivated, adjuvanted]): Extension of indication to include children from 6 months to less than 18 years of age for the prophylaxis of influenza in an officially declared pandemic situation.

Keytruda (pembrolizumab): extension of indication for the first-line treatment, in combination with trastuzumab, fluoropyrimidine and platinum-containing chemotherapy, of locally advanced unresectable or metastatic HER2-positive gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1. Keytruda was already authorised for the treatment of melanoma, non-small cell lung carcinoma, classical Hodgkin lymphoma, urothelial carcinoma, head and neck squamous cell carcinoma, renal cell carcinoma, colorectal cancer, oesophageal carcinoma, breast cancer, endometrial carcinoma, cervical cancer and several microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) cancers.

Opdivo (nivolumab): extension of indication to the adjuvant treatment, as monotherapy, of adults and adolescents 12 years of age and older with Stage IIB or IIC melanoma, or melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection. This adds the treatment of stage IIB and IIC melanoma to the existing indication. Opdivo was furthermore already authorised for the treatment of melanoma, non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell cancer of the head and neck, urothelial carcinoma, colorectal cancer, oesophageal squamous cell carcinoma, and gastric, gastro-oesophageal junction (GEJ) and oesophageal adenocarcinoma.

Spikevax bivalent Original/Omicron BA.4-5 (elasomeran / imelasomeran and elasomeran / davesomeran and elasomeran / COVID-19 mRNA vaccine [nucleoside-modified]): extension of indication to the active immunisation to prevent COVID-19 caused by SARS-CoV-2 in individuals 6 months of age and older. Previously, Spikevax bivalent Original/Omicron BA.4-5 was authorised in individuals 6 years of age and older and only after receiving at least a primary vaccination course against COVID-19.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Briumvi (ublituximab) is indicated for the treatment of adult patients with relapsing forms of multiple sclerosis with active disease defined by clinical or imaging features. EPAR Briumvi.

Columvi (glofitamab) as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, after two or more lines of systemic therapy. EPAR Columvi.

Lytgobi (futibatinib) monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. EPAR Lytgobi.

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase deficiency). EPAR Opfolda.

 

• In vitro diagnostic medical device - To detect rearrangements involving the ALK gene via fluorescence.

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/documents/assessment-report/briumvi-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/columvi-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/lytgobi-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/opfolda-epar-public-assessment-report_en.pdf

Aquipta (atogepant) received a positive opinion for prophylaxis of migraine in adults who have at least 4 migraine days per month. Migraine is a chronic condition characterized by recurrent headaches that may affect quality of life and productivity. Although the causes of migraine are unknown, calcitonin gene-related peptides (CGRPs) have been found to be involved in the development of pain in migraine. Aquipta belongs to the CGRP receptor antagonists, also known as gepants.  Aquipta blocks the binding of CGRPs to their receptor, thereby preventing the development of the cascade of events that result in a migraine attack.

Jesduvroq (daprodustat) received a positive opinion for the treatment of symptomatic anaemia associated with chronic kidney disease (CKD) in adults on chronic maintenance dialysis. Several factors can contribute to the development of anaemia in patients with CKD, including insufficient production of erythropoietin, impaired iron absorption, shorter lifespan of red blood cells or blood loss associated with haemodialysis. Jesduvroq inhibits the hypoxia-inducible factor (HIF) prolyl hydroxylase, thereby stabilizing HIFs’ alpha subunit (HIF-α). The HIF transcription factors subsequently induce the expression of erythropoietin.

Bimatoprost - Indicated for the reduction of intraocular pressure (IOP) in adults with open angle glaucoma or ocular hypertension who are unsuitable for topical IOP-lowering medications.

COVID-19 vaccine - Immunisation to prevent COVID-19 caused by SARS-CoV-2.

Danicopan - Orphan - Treatment of extravascular haemolysis in patients with paroxysmal nocturnal haemoglobinuria.

Flortaucipir (18f) - Indicated for Positron Emission Tomography imaging of the brain.

In vitro diagnostic medical device - Is indicated as an aid in the selection of adult hemophilia A patients for whom valoctocogene roxaparvovec treatment is being considered.

Retifanlimab - Orphan - Treatment of Merkel cell carcinoma.

Serplulimab - Orphan - First-line treatment of adult patients with extensive-stage small cell lung cancer.
 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Akeega (niraparib / abiraterone acetate) is indicated with prednisone or prednisolone for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) and BRCA 1/2 mutations (germline and/or somatic) in whom chemotherapy is not clinically indicated. EPAR Akeega.

Arexvy (Recombinant respiratory syncytial virus pre-fusion F protein, adjuvanted with AS01E) is indicated for active immunisation for the prevention of lower respiratory tract disease caused by respiratory syncytial virus in adults 60 years of age and older. EPAR Arexvy.

Bimervax (COVID-19 Vaccine [recombinant, adjuvanted]) is indicated as a booster for active immunisation to prevent COVID-19 in individuals 16 years of age and older who have previously received a mRNA COVID-19 vaccine. EPAR Bimervax.

Hyftor (sirolimus) is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older. EPAR Hyftor.

Omvoh (mirikizumab) is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic treatment. EPAR Omvoh.

Pedmarqsi (sodium thiosulfate) is indicated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to < 18 years of age with localised, non-metastatic, solid tumours. EPAR Pedmarqsi.

 

Links, in order of appearance:

Committee for Medicinal Products for Human Use (CHMP) (EMA)

European public assessment reports: background and context (EMA)

Assessment report: Akeega (pdf)

Assessment report: Arexvy (pdf)

Assessment report: Bimervax (pdf)

Assessment report: Hyftor (pdf)

Assessment report: Omvoh (pdf)

Assessment report: Pedmarqsi (pdf)

Comirnaty Original/Omicron BA.4-5 (COVID-19 mRNA Vaccine [nucleoside modified]): extension of indication to include the active immunisation to prevent COVID-19 in individuals 6 months of age and older who have not previously received at least a primary vaccination course against COVID-19. Previously, Comirnaty Original/Omicron BA.4-5 was only authorised in patients who had previously received at least a primary vaccination course against COVID-19. The dose is different in the different age groups.

Imjudo (tremelimumab): extension of indication to the first-line treatment of adults with metastatic non-small cell lung cancer with no sensitising EGFR mutations or ALK positive mutations, in combination with durvalumab and platinum-based chemotherapy. Imjudo was already authorised for the treatment of hepatocellular carcinoma.

Jardiance (empagliflozin): extension of indication to the treatment of chronic kidney disease in adults. Jardiance was already authorised for the treatment of type 2 diabetes mellitus and chronic heart failure.

Lonsurf (trifluridine / tipiracil): extension of indication to the treatment, in combination with bevacizumab, of adult patients with metastatic colorectal cancer who have received two prior anticancer treatment regimens including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-VEGF agents, and/or anti-EGFR agent. Lonsurf was already authorised for the treatment of gastric cancer and colorectal cancer as monotherapy.

Mircera (methoxy polyethylene glycol-epoetin beta): extension of indication to include the treatment of symptomatic anaemia associated with chronic kidney disease (CKD) in paediatric patients from 3 months to less than 18 years of age who are converting from another erythropoietin stimulating agent (ESA) after their haemoglobin level was stabilised with the previous ESA. Mircera was already authorised in adults for the treatment of symptomatic anaemia associated with CKD.

Refixia (nonacog beta pegol): extension of indication to include the treatment and prophylaxis of bleeding in patients below 12 years of age with haemophilia B (congenital factor IX deficiency). Refixia was already authorised in patients 12 years of age and older.

Soliris (eculizumab): extension of indication to include the treatment of refractory generalized myasthenia gravis in patients aged 6 years and above who are anti-acetylcholine receptor (AChR) antibody-positive. Soliris was already authorised in this indication in adults. Furthermore, Soliris was already authorised for the treatment of paroxysmal nocturnal haemoglobinuria, atypical haemolytic uremic syndrome and neuromyelitis optica spectrum disorder.

Trodelvy (sacituzumab govitecan): extension of indication to the treatment as monotherapy of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer who have received endocrine-based therapy, and at least two additional systemic therapies in the advanced setting. Trodelvy was already authorised for the treatment of triple-negative breast cancer.

Pylclari (piflufolastat (18F)) received a positive opinion for the detection of prostate-specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer (PCa) in the following clinical settings:

- Primary staging of patients with high-risk PCa prior to initial curative therapy,

- To localize recurrence of PCa in patients with a suspected recurrence based on increasing serum prostate-specific antigen (PSA) levels after primary treatment with curative intent.

Prostate cancer is the second most common cancer in men. The membrane protein PSMA is expressed in the prostate in high levels and further overexpressed in prostate cancer. Pylclari is a radioactive diagnostic agent that binds to PSMA and is labelled with a positron-emitting isotope (18F) used for imaging by PET. Therefore, by binding to PSMA, the radiopharmaceutical will be able to detect prostate cancer cells.

Pylclari is the second PSMA-targeted radiopharmaceutical for prostate cancer detection via PET authorised in the EU (after Locametz (gozetotide), authorised in the EU since December 2022).

Ztalmy (ganaxolone) received a positive opinion for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

CDD is a rare X-linked dominant genetic disease caused by loss-of-function mutations in the CDKL5 gene. CDKL5 is involved in brain development and function and patients with CDD present with epilepsy and severe developmental delay, including cognitive and motor impairment. Ztalmy is a synthetic analogue of allopregnanolone, a naturally-occurring neurosteroid that positively modulates the gamma-aminobutyric acid type A (GABAA) receptor. Ztalmy exerts its anti-seizure effects by allosterically modulating GABAA receptors.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Elfabrio (pegunigalsidase alfa) is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). EPAR Elfabrio.

Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase deficiency).  EPAR Pombiliti.

Tibsovo (ivosidenib) in combination with azacitidine is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy.

Tibsovo monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. EPAR Tibsovo.

Vafseo (vadadustat) is indicated for the treatment of symptomatic anaemia associated with chronic kidney disease in adults on chronic maintenance dialysis. EPAR Vafseo.

Opdivo (nivolumab): extension of indication to the neoadjuvant treatment of resectable non-small cell lung cancer at high risk of recurrence in adult patients whose tumours have PD-L1 expression ≥ 1%, in combination with platinum-based chemotherapy. Opdivo was already authorised for the treatment of melanoma, non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell cancer of the head and neck, urothelial carcinoma, colorectal cancer, oesophageal squamous cell carcinoma, and gastric, gastro-oesophageal junction and oesophageal adenocarcinoma.

Sogroya (somapacitan): extension of indication for the replacement of endogenous growth hormone (GH) in children aged 3 years and above and adolescents with growth failure due to GH deficiency (paediatric GHD). Sogroya was already authorised in adults.

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Aprocitentan - Treatment of resistant hypertension.

Buprenorphine - Treatment of opioid drug dependence.

In vitro diagnostic medical device - To determine Human Epidermal Growth Factor Receptor 2 (HER2) oncoprotein status.

Methylphenidate hydrochloride - PUMA - Treatment of Attention Deficit Hyperactivity Disorder (ADHD) in children aged 6 years of age and over.

rdESAT-6 + rCFP-10 - Diagnosis of infection with Mycobacterium tuberculosis.

Sugemalimab - Treatment of adults with metastatic non-small cell lung cancer.

 

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/medicines/human/EPAR/locametz

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/documents/assessment-report/elfabrio-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/pombiliti-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/tibsovo-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/vafseo-epar-public-assessment-report_en.pdf

 

​​​​​​

Bevacizumab - Treatment of neovascular (wet) age-related macular degeneration.

Cefepime / enmetazobactam - Treatment of:

complicated urinary tract infections (including pyelonephritis);

hospital-acquired pneumonia (HAP), including ventilator associated pneumonia;

patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above and

infections due to aerobic Gram-negative organisms in adults with limited treatment options.

Concizumab - Routine prophylaxis to prevent or reduce the frequency of bleeding in patients with:

haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors ≥ 12 years of age;

haemophilia B (congenital factor IX deficiency) with FIX inhibitors of any age.

Elranatamab - Orphan - Treatment of adult patients with relapsed or refractory multiple myeloma.

Exagamglogene autotemcel - ATMP - Treatment of transfusion-dependent β-thalassemia and sickle cell disease.

Germanium (68ge) chloride / gallium (68ga) chloride - In vitro radiolabelling of specific carrier molecules to be used for positron emission tomography imaging.

Influenza virus a/turkey/turkey/1/2005 (h5n1) nibrg-23 strain, ha surface antigen - Prophylaxis of influenza.

Lecanemab - A disease modifying treatment in adult patients with Mild Cognitive Impairment due to Alzheimer’s disease and Mild Alzheimer’s disease (Early Alzheimer’s disease)

Omaveloxolone - Orphan - Treatment of Friedreich’s ataxia.

Omecamtiv mecarbil - Treatment of adult patients with symptomatic chronic heart failure and reduced ejection fraction less than 30%.

Pandemic influenza vaccine (surface antigen, inactivated, adjuvanted) - Active immunisation for the prevention of disease caused by the influenza A virus H5N1 subtype contained in the vaccine.

Pegcetacoplan - Treatment of geographic atrophy secondary to age-related macular degeneration.

Respiratory syncytial virus vaccine - Prevention of respiratory tract disease.

Talquetamab - Orphan - Monotherapy treatment of adult patients with relapsed and refractory multiple myeloma.

 

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/news/first-vaccine-protect-older-adults-respiratory-syncytial-virus-rsv-infection

https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation

https://www.ema.europa.eu/en/glossary/hybrid-medicine

https://www.ema.europa.eu/en/medicines/human/EPAR/zavesca

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

https://www.ema.europa.eu/documents/assessment-report/opzelura-epar-public-assessment-report_en.pdf

 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Opzelura (ruxolitinib) is indicated for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.  EPAR Opzelura.

Adempas (riociguat): extension of indication to the treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class II to III in combination with endothelin receptor antagonists. Adempas was already authorised for the treatment of PAH and chronic thromboembolic pulmonary hypertension in adults.

Bimzelx (bimekizumab) has received a positive opinion for the following extension of indications:

For the treatment of adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation as indicated by elevated C-reactive protein and/or magnetic resonance imaging (MRI) who have responded inadequately or are intolerant to non-steroidal anti-inflammatory drugs;

For the treatment of adults with active ankylosing spondylitis who have responded inadequately or are intolerant to conventional therapy; and

For the treatment, alone or in combination with methotrexate, of active psoriatic arthritis in adults who have had an inadequate response or who have been intolerant to one or more disease-modifying antirheumatic drugs.

Bimzelx was already authorised for the treatment of plaque psoariasis.

Cosentyx (secukinumab): extension of indication to the treatment of active moderate to severe hidradenitis suppurativa (acne inversa) in adults with an inadequate response to conventional systemic hidradenitis suppurativa therapy. Cosentyx was already authorised for the treatment of plaque psoriasis, psoriatic arthritis, axial spondyloarthritis and juvenile idiopathic arthritis.

Opdivo (nivolumab): extension of indication to adolescents 12 years of age and older:

For the treatment of advanced (unresectable or metastatic) melanoma, as monotherapy or in combination with ipilimumab;

For the adjuvant treatment of melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection, as monotherapy.

Opdivo was already authorised in these indications in adults. Furthermore, Opdivo was already authorised for the treatment of non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell cancer of the head and neck, urothelial carcinoma, mismatch repair deficient or microsatellite instability-high colorectal cancer, oesophageal squamous cell carcinoma, oesophageal or gastro-oesophageal junction (GEJ) cancer and gastric, GEJ or oesophageal adenocarcinoma.

Orkambi (lumacaftor / ivacaftor): extension of indication for Orkambi granules to the treatment of cystic fibrosis in patients from 1 year of age who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Orkambi granules was already authorised in patients aged 2 and older.

Revestive (teduglutide): extension of indication to the treatment of patients 4 months corrected gestational age and above with Short Bowel Syndrome. Patients should be stable folllowing a period of intestinal adaptation after surgery. Revestive was already authorised for patients aged 1 year and older.

Ronapreve (casirivimab / imdevimab): extension of indication to the treatment of COVID-19 in adults and adolescents aged 12 years and older weighing at least 40 kg and receiving supplemental oxygen, who have a negative SARS-CoV-2 antibody test result. Ronapreve was already authorised for the prevention of COVID-19 and for treatment of COVID-19 in patients who do not require supplemental oxygen and who are at increased risk of progressing to severe COVID-19.

Spikevax bivalent Original/Omicron BA.4-5 (elasomeran): extension of indication to the active immunisation to prevent COVID-19 in individuals 6 years of age and older who have previously received at least a primary vaccination course against COVID-19. Spikevax bivalent Original/Omicron BA.4-5 was already authorised in patients aged 12 years of age and older.

Vemlidy (tenofovir alafenamide): extension of indication to the treatment of chronic hepatitis B (CHB) in adults and paediatric patients 6 years of age and older weighing at least 25 kg. Vemlidy was already authorised in adolescents aged 12 years and older.

Yervoy (ipilimumab): extension of indication to the treatment of adolescents 12 years of age and older with advanced (unresectable or metastatic) melanoma, in combination with nivolumab. Yervoy was already authorised in this indication as monotherapy. Furthermore, Yervoy was also already authorised for the treatment of renal cell carcinoma, non-small cell lung cancer, malignant pleural mesothelioma, mismatch repair deficient or microsatellite instability-high colorectal cancer and oesophageal squamous cell carcinoma.

Arexvy (Respiratory Syncytial Virus (RSV) vaccine [recombinant, adjuvanted]) is indicated for active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus in adults 60 years of age and older.

RSV is a single-stranded RNA virus that infects people of all ages but does not confer long-term immunity. In vulnerable individuals, such as older adults, re-infections could lead to more severe manifestations of the disease, such as LRTD. Arexvy includes an engineered version of the well conserved RSV F surface glycoprotein, which is required for the entry of the virus into the cell.

Arexvy is the first vaccine authorised to protect adults from RSV. For more information see the EMA news announcement  on Arexvy.

Camzyos (mavacamten) is indicated for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients.

HCM is a disease in which the heart muscle becomes thickened, impairing the blood-pumping and electrical conduction function of the heart. HCM is classified as obstructive when the outflow of blood from the left ventricle is obstructed. Camzyos selectively, reversibly and allosterically inhibits cardiac myosin, which is responsible for the contraction of the heart.

Columvi (glofitamab) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy.

DLBCL is an aggressive cancer of the B-lymphocytes and it is the most common form of non-Hodgkin lymphoma. As many mature B-cell lymphomas, DLBCL express the surface antigen CD20. Columvi is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Columvi binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated B-cell lysis.

Jaypirca (pirtobrutinib) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton’s tyrosine kinase (BTK) inhibitor.

MCL is rare type of non-Hodgkin’s lymphoma. BTK is a soluble tyrosine kinas involved in the development and maturation of B cells, which has been shown to play an important role in the development of various B-cell cancers, such as MCL. Jaypirca is a highly selective small molecule non-covalent BTK inhibitor.

Lytgobi (futibatinib) received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

CCA is a rare cancer of the bile duct with high genomic diversity. Recent next-generation sequence analyses have shown that many mutations typically found in other cancer types are also present in CCA, including fusions and rearrangements in FGFR2. Indeed, fusions in the FGFR2 gene have been found in around 10 to 15 % of intrahepatic CCA. Lytgobi is a small molecule covalent tyrosine kinase inhibitor that irreversibly binds to and inhibits FGFR1/2/3/4.

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase deficiency).

Pompe disease is an autosomal recessive metabolic disease caused by a reduced or absent activity of the lysosomal alpha-glucosidase. As a consequence, glycogen accumulates in lysosomes, causing damage in the muscle (including heart) and nerve cells throughout the body. Opfolda has to be administered in combination with Pombiliti (cipaglucosidase alfa), a recombinant human alpha-glucosidase intended for enzyme replacement, which is conjugated with synthetic mannose 6-phosphate-rich glycans to enhance receptor targeting. Opfolda is an enzyme stabiliser that improves stability of cipaglucosidase in the bloodstream.

Opfolda is a hybrid medicine of Zavesca (miglustat), which is authorised in the EU since November 2002 for the treatment of type 1 Gaucher disease. Although both contain the same active substance, miglustat, Opfolda has a lower strength and a different indication.

 

• Aumolertinib - Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer.
• Dopamine hydrochloride - PUMA - Treatment of hypotension in neonates, infants and children.
• Etrasimod - Treatment of patients with moderately to severely active ulcerative colitis.
• In vitro diagnostic medical device - To detect internal tandem duplication and tyrosine kinase domain mutations D835 and I836 in the FLT3 gene.
• Momelotinib - Orphan - Treatment of disease-related splenomegaly or symptoms and anaemia.
• Rozanolixizumab - Orphan - Treatment of generalised myasthenia gravis.
• Teriparatide - Orphan - Parathormone replacement therapy indicated for the treatment of hypoparathyroidism in adults.
• Tofersen - Treatment of adults with amyotrophic lateral sclerosis associated with a mutation in the superoxide dismutase 1 gene.
• Toripalimab - Combination treatment for metastatic or recurrent locally advanced nasopharyngeal carcinoma and for metastatic or recurrent oesophageal squamous cell carcinoma.

 

Links, in order or appearance:

https://www.ema.europa.eu/en/committees/committee-medicinal-products-human-use-chmp

https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/paediatric-medicines/paediatric-use-marketing-authorisations

https://www.ema.europa.eu/en/medicines/what-we-publish-when/european-public-assessment-reports-background-context

Bimervax (COVID-19 Vaccine [recombinant, adjuvanted]) is indicated as a booster for active immunisation to prevent COVID-19 in individuals 16 years of age and older who have previously received an mRNA COVID-19 vaccine.

COVID-19 or Coronavirus disease 2019, is a contagious disease caused by the Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Although this virus can infect a wide variety of cells, it is most known for causing respiratory tract symptoms, which vary from mild to severe. The virus enters the cells mainly by binding to the angiotensin converting enzyme 2 (ACE2). Bimervax is a vaccine harbouring a recombinant modified Spike protein antigen. The Spike protein is the major viral surface protein and it mediates viral entry by binding to ACE2 on the host cell. Bimervax is the eighth vaccine recommended in the European Union for protecting against COVID-19. However, it has only received a positive opinion as a booster and not for primary vaccination. For more information visit the EMA news announcement on Bimervax.

Briumvi (ublituximab) is indicated for the treatment of adult patients with relapsing forms of multiple sclerosis with active disease defined by clinical or imaging features.

Multiple sclerosis is a chronic neurodegenerative autoimmune disease of the central nervous system, which leads to irreversible decrease in physical and cognitive function. Briumvi is an immunosuppressant monoclonal antibody, which binds to the CD20 surface antigen of B-cells, leading to antibody-dependent cell death.

 

Omvoh (mirikizumab) is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic treatment.

UC is a chronic inflammatory disease that affects the colon. Although the aetiology is not fully understood, many factors seem to be involved in the pathogenesis of the disease, including altered immune responses, environmental and genetics factors. Omvoh is a monoclonal antibody that binds to interleukin 23 (IL-23), a pro-inflammatory cytokine involved in promoting inflammation in UC, and inhibits its binding to the IL23 receptor, thereby disrupting the cytokine signalling cascade.

Pedmarqsi (sodium thiosulfate) received a positive opinion for a paediatric-use marketing authorisation (PUMA) is indicated for the prevention of ototoxicity induced by cisplatin chemotherapy in patients 1 month to < 18 years of age with localised, non-metastatic, solid tumours. Cisplatin is a platinum-based chemotherapeutic agent widely used for the treatment of solid tumours. However, the development of ototoxicity is a serious hindrance of effective treatment. Although the mechanism for this toxicity has not been fully elucidated, it is accepted that the excessive production of reactive oxygen species (ROS) plays a major role in the development of platinum-induced hearing loss. Pedmarqsi is an otoprotectant with free-radical scavenging potential, which directly reduced ROS. Furthermore, it can bind to platinum, producing a biologically-inactive complex.

 

https://www.ema.europa.eu/en/news/ema-recommends-approval-bimervax-covid-19-booster-vaccine

 

Breyanzi (lisocabtagene maraleucel): extension of indication to the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL), high grade B cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), who relapsed within 12 months from completion of, or are refractory to, first-line chemoimmunotherapy. Breyanzi was already authorised for the treatment of relapsed or refractory DLBCL, PMBCL and FL3B, after two or more lines of systemic therapy.

Entresto and Neparvis (sacubitril / valsartan): extension of indication to include the treatment of symptomatic chronic heart failure with left ventricular systolic dysfunction in children and adolescents aged one year or older. Entresto and Neparvis were already authorised in adults.

Tenkasi (oritavancin): extension of indication to the treatment of acute bacterial skin and skin structure infections in paediatric patients aged 3 months and older. Tenkasi was already authorised in adults.

Ultomiris (ravulizumab): extension of indication to the treatment of adult patients with neuromyelitis optica spectrum disorder who are anti-aquaporin 4 (AQP4) antibody-positive. Ultomiris was already authorised for the treatment of paroxysmal nocturnal haemoglobinuria, atypical haemolytic uremic syndrome and generalized myasthenia gravis.

Wegovy (semaglutide). Extension of indication, as an adjunct to a reduced-calorie diet and increased physical activity, for weight management in adolescents ages 12 years and above with obesity and body weight above 60 kg. Wegovy was already authorised in adults.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Imjudo (tremelimumab) in combination with durvalumab is indicated for the first line treatment of adults with advanced or unresectable hepatocellular carcinoma. EPAR Imjudo.

Sotyktu (deucravacitinib) is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. EPAR Sotyktu.

Tremeliumab AstraZeneca (tremelimumab) in combination with durvalumab and platinum-based chemotherapy is indicated for the first-line treatment of adults with metastatic non-small cell lung cancer with no sensitising EGFR mutations or ALK positive mutations. EPAR Tremelimumab AstraZeneca.

 

https://www.ema.europa.eu/en/documents/assessment-report/imjudo-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/tremelimumab-astrazeneca-epar-public-assessment-report_en.pdf

https://www.ema.europa.eu/documents/assessment-report/sotyktu-epar-public-assessment-report_en.pdf

 

Akeega (niraparib / abiraterone acetate) is indicated with prednisone or prednisolone for the treatment of adult patients with metastatic castration resistant prostate cancer (mCRPC) and BRCA1/2 gene mutations (germline and/or somatic) in whom chemotherapy is not clinically indicated.

Prostate cancer is the second most common cancer in men globally and, although the overall survival for patients with localised cancer is very high, the life expectancy decreases dramatically for patients with advanced disease. Abiraterone acetate is a precursor of abiraterone, an antiandrogen that decreases testosterone synthesis by inhibiting CYP17.

Niraparib is a poly ADP ribose polymerase (PARP) inhibitor. PARPs are involved in many cellular processes including DNA repair and stability and apoptosis. Niraparib blocks DNA-associated PARPs, thereby inhibiting DNA repair and promoting the formation of double strand breaks in replicating cells. While in normal cells these can be repaired by homologous recombination, the absence of functional BRCA1/2 leads to the activation of error-prone repair pathways, ultimately leading to genome instability and cell death.

Elfabrio (pegunigalsidase alfa) is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). Fabry disease is a rare X-linked genetic disorder caused by mutations in the alpha-galactosidase A gene (GLA), which result in a decreased activity of the GLA enzyme. GLA is involved in many catabolic pathways, including the processing of glycoproteins and glycolipids. A reduction in its activity leads to accumulation of glycosphingolipids (mainly globotriaosylceramide, Gb3) within lysosomes in various tissues, resulting in swelling and endothelial proliferation. The clinical manifestations of Fabry disease include severe pain in the limbs, skin lesions, eye opacities and deterioration of kidney and heart function. Elfabrio is a recombinant human GLA intended as enzyme-replacement therapy.

Hyftor (sirolimus) is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and paediatric patients aged 6 years and older. TSC is a rare autosomal dominant disease caused by loss-of-function mutations in the genes TSC1 or TSC2, encoding the proteins hamartin and tuberin, respectively. The disease is characterised by the presence of non-cancerous tumours, hamartomas, which appear in many vital organs including the brain, kidneys, heart and skin. This unregulated cell proliferation results from the hyperactivation of mammalian target of rapamycin (mTOR), normally suppressed by hamartin and tuberin. Angiofibromas are one type of skin manifestation of TSC and it is present in a great majority of patients.

Hyftor is an mTOR inhibitor and contains the same active substance as Rapamune, which has been authorised in the EU since 13 March 2001. Rapamune is authorised in the EU for prophylaxis of kidney transplant rejection and for the treatment of sporadic lymphangioleiomyomatosis. Although both contain the same active substance (sirolimus or rapamycin), Hyftor is available as a gel intended for local administration while Rapamune is available as an oral solution.

Opzelura (ruxolitinib) is indicated for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age. Non-segmental, or generalised, vitiligo is an autoimmune disorder that manifests as acquired and progressive depigmented patches of the skin, mucosa and hair, caused by a selective loss of melanocytes. Although its aetiology is unknown, it is likely that stress, genetic, metabolic and environmental factors play a role in the onset of vitiligo.

Furthermore, an interferon-gamma-mediated immune response via the activation Janus kinase/signal transducers and activators of transcription (JAK/STAT) signalling pathway has been linked to the pathogenesis of vitiligo.

Opzelura is a small molecule JAK inhibitor intended for topic administration and it is the first authorised treatment for this disorder. Ruxolitinib is also the active substance in the medicinal product Jakavi, authorised in the EU since August 2012 and intended for the treatment of myelofibrosis, polycythaemia vera and graft-versus-host disease.

Tibsovo (ivosidenib) in combination with azacitidine is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy.

Furthermore, Tibsovo (ivosidenib) received a positive opinion for a second indication this month for the treatment, as monotherapy, of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy.

AML is a cancer of the white blood cells characterised by the overproduction of immature myeloid cells and mutations in IDH can be found in up to 30% of AML patients.

CCA is a cancer of the bile duct with high genomic diversity. Recent next-generation sequence analyses have shown that many mutations typically found in other cancer types are also present in CCA, including mutations in IDH. Indeed, mutations in IDH have been found in around 15 to 20 % of intrahepatic CCA.

IDH1 is an enzyme of the tricarboxylic acid cycle responsible for the oxidative decarboxylation of isocitrate to α-ketoglutarate. Mutations in IDH lead to the accumulation of 2-hydroxyglutarate (2-HG), which affects numerous α-ketoglutarate-dependent processes such as histone and DNA methylation, thereby promoting malignant transformation. Tibsovo is first-in-class small molecule inhibitor of IDH1, exerting its effect by decreasing the levels of 2-HG.

Vafseo (vadadustat) is indicated for the treatment of symptomatic anaemia associated with chronic kidney disease (CKD) in adults on chronic maintenance dialysis. Several factors can contribute to the development of anaemia in patients with CKD, including insufficient production of erythropoietin, impaired iron absorption, shorter lifespan of red blood cells or blood loss associated with haemodialysis. Vafseo inhibits the hypoxia-inducible factor (HIF) prolyl hydroxylase, thereby stabilizing HIFs, which induce the expression of erythropoietin.

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Hemgenix (etranacogene dezaparvovec) is indicated for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. EPAR Hemgenix.

Spevigo (spesolimab) is indicated for the treatment of flares in adult patients with generalised pustular psoriasis as monotherapy. EPAR Spevigo.

Esbriet (pirfenidone): extension of indication to the treatment of idiopathic pulmonary fibrosis (IPF) in adults. Esbriet was previously only authorised for the treatment of mild to moderate IPF.

Libtayo (cemiplimab): extension of indication to include, in combination with platinum‐based chemotherapy, the first-line treatment of adult patients with non-small cell lung cancer (NSCLC) expressing PD-L1 (in ≥ 1% of tumour cells), with no EGFR, ALK or ROS1 aberrations, who have locally advanced NSCLC who are not candidates for definitive chemoradiation, or metastatic NSCLC.

Libtayo as monotherapy was already authorised for the first-line treatment of adult patients with NSCLC expressing PD-L1 (in ≥ 50% tumour cells), with no EGFR, ALK or ROS1 aberrations, who have locally advanced NSCLC and who are not candidates for definitive chemoradiation, or metastatic NSCLC.

Furthermore, Libtayo was already authorised for the treatment of cutaneous squamous cell carcinoma, basal cell carcinoma and cervical cancer.

Rinvoq (upadacitinib): extension of indication to the treatment of adult patients with moderately to severely active Crohn’s disease who have had an inadequate response, lost response or were intolerant to either conventional therapy or a biologic agent. Rinvoq was already authorised for the treatment of rheumatoid and psoriatic arthritis, axial spondyloarthritis, atopic dermatitis and ulcerative colitis.

TachoSil (human fibrinogen / human thrombin): extension of indication to the supportive treatment of children from 1 month of age in surgery for improvement of haemostasis, to promote tissue sealing and for suture support in vascular surgery where standard techniques are insufficient. TachoSil was already authorised in adults in this indication and for supportive sealing of the dura mater to prevent postoperative cerebrospinal leakage following neurological surgery.

Update on the following authorised medicines for the prevention of COVID-19: The CHMP has recommended authorising the use of COVID-19 Vaccine Valneva (inactivated, adjuvanted) as a booster dose for adults 18 to 50 years of age.

Recently started procedures:

• Cabotegravir - Pre-exposure prophylaxis of HIV-1 infection.
• Epcoritamab - Orphan - Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma.
• Epinephrine - Treatment of allergic reactions (anaphylaxis) and idiopathic or exercise induced anaphylaxis.
• Lebrikizumab - Treatment of moderate-to-severe atopic dermatitis in adults and adolescents.
• Leniolisib - Orphan - Treatment of activated phosphoinositide 3-kinase delta syndrome.
• Vamorolone - Orphan - Treatment of Duchenne muscular dystrophy.

Sotyktu (deucravacitinib) is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. Psoriasis is a common chronic inflammatory skin disease with different clinical phenotypes with 80 to 90 % of patients being affected by plaque psoriasis. Raised, sharply demarcated, erythematous plaques covered by silvery scales characterize plaque psoriasis. Sotyktu is a small molecule highly selective inhibitor of the tyrosine kinase 2 (TYK2), a member of the Janus kinase (JAK) protein family, a family of kinases involved in cytokine signalling. By inhibiting TYK2, Sotyktu reduces inflammatory cascade reactions.

Byfavo (remimazolam): extension of indication to the induction and maintenance of general anesthesia in adults. Byfavo was already authorised for procedural sedation in adults.

Dupixent (dupilumab): extension of indication to the treatment of severe atopic dermatitis (AD) in children 6 months to 11 years old who are candidates for systemic therapy. Dupixent was already authorised in children 6 to 11 years old in this setting and in adults and adolescents older than 12 year for the treatment of moderate-to-severe AD. Furthermore, Dupixent was already authorised for the treatment of asthma, chronic rhinosinusitis with nasal polyposis and prurigo nodularis.

Nubeqa (darolutamide): extension of indication to the treatment of adult men with metastatic hormone sensitive prostate cancer in combination with docetaxel and androgen deprivation therapy. Nubeqa was already authorised for the treatment of non-metastatic castration resistant prostate cancer.

Reblozyl (luspatercept): extension of indication to the treatment of anaemia associated with nontransfusion-dependent beta-thalassaemia in adults. Reblozyl was already authorised for the treatment of anaemia associated with transfusion-dependent beta-thalassaemia.

Trecondi (treosulfan): extension of indication to include the conditioning treatment prior to allogeneic haematopoietic stem cell transplantation in paediatric patients older than one month with nonmalignant diseases, in combination with fludarabine. Trecondi was already authorised for conditioning treatment in adults with malignant and non-malignant diseases and in paediatric patients with malignant diseases.

Trulicity (dulaglutide): extension of indication to the treatment of patients 10 years and above with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise:

• as monotherapy when metformin is considered inappropriate due to intolerance or contraindications
• in addition to other medicinal products for the treatment of diabetes.

Trulicity was already authorised in adults in this indication.

Wakix (pitolisant): extension of indication to the treatment of narcolepsy with or without cataplexy in adolescents and children from the age of 6 years. Wakix was already authorised in adults in this indication.

Recently started procedures:

• Dabrafenib - Orphan - Treatment of glioma.
• Trametinib - Orphan - Treatment of glioma.
• Fezolinetant - Treatment of moderate to severe vasomotor symptoms associated with
• menopause.
• Latanoprost - Reduction of elevated intraocular pressure.
• Zilucoplan - Orphan - treatment of generalised myasthenia gravis in adults.

EMAs website: Committee for Medicinal Products for Human Use (CHMP)

EMAs website: European public assessment reports: background and context 

The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:

Ebvallo (tabelecleucel) is indicated as monotherapy for treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. Ebvallo is an advanced therapy medicinal product (ATMP) composed of allogeneic T-cells, selected for their specific immunological function (lysis of EBV+ targets) and sufficient human leukocyte antigen
(HLA) similarity between donor and recipient. Although limited to a niche indication, the use of allogenic T-cells is a new, previously unauthorized mode of action for the immunotherapy of cancer. EPAR Ebvallo.

Eladynos (abaloparatide) is indicated for the treatment of osteoporosis in postmenopausal women at increased risk of fracture. EPAR Eladynos.

Locametz (gozetotide), after radiolabelling with gallium-68, is indicated for the detection of prostatespecific membrane antigen (PSMA)-positive lesions with positron emission tomography in adults with prostate cancer (PCa) in the following clinical settings:

• Primary staging of patients with high-risk PCa prior to primary curative therapy,
• Suspected PCa recurrence in patients with increasing levels of serum prostate-specific antigen (PSA) after primary curative therapy,
• Identification of patients with PSMA-positive progressive metastatic castration-resistant prostate cancer (mCRPC) for whom PSMA-targeted therapy is indicated.

EPAR Locametz.

Mycapssa (octreotide) is indicated for maintenance treatment in adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues. EPAR Mycapssa.

Pluvicto [lutetium-177 vipivotide tetraxetan], in combination with androgen deprivation therapy with or without androgen receptor pathway inhibition is indicated for the treatment of adult patients with progressive PSMA-positive mCRPC who have been treated with androgen receptor pathway inhibition and taxane-based chemotherapy. While PSMA has been used in the diagnostic of prostate cancer for several years, Pluvicto is the first cancer treatment targeting PSMA that received a positive CHMP opinion. EPAR Pluvicto.

Zynlonta (loncastuximab tesirine), as monotherapy, is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma, after two or more lines of systemic therapy. EPAR Zynlonta.

Links, in order or appearance:

EMA: Assessment report, Ebvallo International non-proprietary name: tabelecleucel (pdf)

EMA: Assessment report, Eladynos International non-proprietary name: abaloparatide (pdf)

EMA: Assessment report, Locametz International non-proprietary name: gozetotide (pdf)

EMA: Assessment report, Mycapssa International non-proprietary name: octreotide (pdf)

EMA: Assessment report, Pluvicto International non-proprietary name: lutetium (177lu) vipivotide tetraxetan (pdf)

EMA: Assessment report, Zynlonta International non-proprietary name: loncastuximab tesirine (pdf)