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FDA Grants Priority Review to Eliglustat (Cerdelga) for Gaucher Disease
The New Drug Application (NDA) for eliglustat (Cerdelga, Sanofi/Genzyme), an investigational oral therapy for adult patients with Gaucher disease type 1, has been granted a 6-month priority review designation by the FDA.
The twice-daily capsule was developed to provide an oral treatment alternative for adults with Gaucher disease type 1. The drug’s clinical development program includes approximately 400 patients treated in 29 countries.
The marketing application for eliglustat is based on positive results from two phase III studies: ENGAGE, which included patients new to therapy, and ENCORE, which included patients switching from enzyme-replacement therapy. The FDA submission also includes 4 years of safety and efficacy data from a phase II trial of eliglustat.
A priority review designation means that the FDA’s goal is to take action on an application within 6 months compared with the 10 months that are required for a standard review.
Patients with Gaucher disease do not have enough of the enzyme beta-glucosidase (glucocerebrosidase), which leads to the accumulation of its substrate, glucosylceramide. As a result, lipid-engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver, and bone marrow. The accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease, and other signs and symptoms. The most common form of Gaucher disease (type 1) generally does not affect the brain.
Eliglustat is an investigational oral glucosylceramide analog. It was designed to partially inhibit the enzyme glucosylceramide synthase, resulting in the reduced production of glucosylceramide.
Source: Sanofi; December 11, 2013.