Fimea published a rapid review of orphan medicinal product sebelipase alfa

Fimea's Assessment of Pharmacotherapies process has published a rapid review that explores the therapeutic and economic effects of sebelipase alfa in the treatment of lysosomal acid lipase deficiency.

Lysosomal acid lipase deficiency

Lysosomal acid lipase deficiency is an extremely rare lipid metabolism disorder, of which two forms of disease are known: the rapidly progressive infantile-onset form (Wolman disease) that typically presents in the first weeks of life, and the later-onset form that presents later. If untreated, the infantile-onset form of the disease usually leads to death before the age of one year. In the later-onset form of the disease, the life expectancy may be normal, but the symptoms, severity and prognosis of the disease vary between individuals. The sebelipase alfa enzyme replacement therapy is currently the only specific treatment affecting the course of the disease in patients with the infantile and later-onset form of the disease.

Fimea estimates that one child with the infantile-onset form of the disease may born in Finland every three years. In Fimea’s estimate, the number of people with the later-onset form of the disease is 2–56 people.

Therapeutic effects

Evidence on the effects of sebelipase alfa in the treatment of the infantile-onset disease is mainly based on one single-arm VITAL study with a small number of patients (n = 9). Six of the patients (67%) reached the age of one year and five (56 %) were still alive at the age of two years. Of the patients in the historical comparison cohort (n = 26), only one (4%) reached the age of one year.

In the treatment of patients with the later-onset disease, the effects of sebelipase alfa have been compared with placebo in an ARISE study (n = 66). The evidence is beneficial compared to placebo, but it is only based on surrogate outcomes (e.g. changes in laboratory values). The effects of the treatment on the patients’ survival or manifestation of hepatic and cardiovascular diseases in this form of the disease are not known.

Costs

Sebelipase alfa treatment is extremely expensive. In the treatment of the infantile-onset disease, the total annual medicine and dosage costs of a single patient are approximately EUR 0.5–1.4 million if the weight of the patient is not more than 20 kg. The annual costs per patient of the later-onset form of the disease are approximately EUR 0.2-0.9 million if the weight of the patient is not more than 80 kg. The large variation in the costs per patients are due to the weight of the patient, the need for a single dose (1–3 mg/kg) and the dosing frequency (every 1–2 weeks). The proportion of drug wastage in the costs can be considerably high in certain cases.

COHERE Finland to issue a recommendation in the spring of 2018

The rapid review was prepared at the request of the Council for Choices in Health Care in Finland (COHERE Finland). During the spring of 2018, COHERE Finland will issue a statement on whether the treatment of lysosomal acid lipase deficiency with sebelipase alfa is to be included in the range of healthcare services financed from public funds. COHERE Finland will announce details on the preparation of its recommendation separately.

Comments

Fimea’s rapid review is open to comments until 28th February 2018. The comments should be sent to the registry office of the Finnish Medicines Agency Fimea at [email protected]. All comments are public and may be published on the Fimea website.

Read more

Rapid review report: Sebelipaasi alfa lysosomaalisen happaman lipaasin puutoksen hoidossa (in Finnish, abstract available in English and in Swedish)