Genzyme announced that the FDA has granted a 6-month Priority Review designation to its New Drug Application (NDA) for Cerdelga (eliglustat), an investigational oral therapy for adults with Gaucher disease type 1. Eliglustat is a novel ceramide analog designed to partially inhibit the enzyme glucosylceramide synthase, resulting in reduced production of glucosylceramide.
The submission for Cerdelga are based on two positive Phase 3 studies for eliglustat, ENGAGE, which included patients new to therapy, and ENCORE which included patients switching from enzyme replacement therapy. The submissions also include four years of safety and efficacy data from the eliglustat Phase 2 study.
Gaucher disease is an inherited condition that causes the accumulation of the substrate glucosylceramide. As a result, lipid engorged cells (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.
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