Cerdelga filed at FDA for Gaucher disease Type 1-Sanofi/Genzyme
Sanofi and its subsidiary Genzyme announced that the FDA has granted a six-month Priority Review designation to its New Drug Application (NDA) for Cerdelga (eliglustat), an investigational oral therapy for adult patients with Gaucher disease type 1. The European Medicines Agency in late October 2013 validated Genzyme�s marketing authorization application (MAA) for eliglustat in the EU.
The marketing applications for Cerdelga are based on two positive Phase III 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 II study.
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