Sanofi and its subsidiary, Genzyme, report that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending approval of Cerdelga (eliglustat) capsules, an oral treatment for certain adults living with Type I Gaucher disease. The European Commission is expected to make a final decision on granting marketing authorization for Cerdelga in the European Union in the coming months.

Cerdelga was approved by the US Food and Drug Administration in August 2014 and is under review by other regulatory authorities.

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, beta-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver, and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease, and a number of other signs and symptoms. Cerdelga is a glucosylceramide analog given orally, which was designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease.

Source: Sanofi