Genzyme Gaucher Treatment Gets Priority Review

Genzyme, a Sanofi company, received a six-month Priority Review designation from the FDA for its NDA for Cerdelga, an investigational oral therapy for adult patients with Gaucher disease type 1.

Genzyme is developing eliglustat, a capsule to be taken twice daily, to provide an effective oral treatment alternative for adult patients with Gaucher disease type 1, and to provide a broader range of treatment options for Gaucher patients and physicians. Genzyme’s clinical development program for eliglustat represents the largest clinical program ever conducted in this orphan treatment area, with approximately 400 patients treated in 29 countries.

The marketing application for Cerdelga is based on two positive Phase III studies for eliglustat, one that included patients new to therapy, and another that included patients switching from enzyme replacement therapy. The submissions also include four years of safety and efficacy data from the drug’s Phase II study.