, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the Food and Drug Administration (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. As previously announced, the European Medicines Agency in late October validated Genzyme’s marketing authorization application (MAA) for eliglustat in the EU.
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 Gaucher disease, with approximately 400 patients treated in 29 countries.
“The acceptance of our applications for Cerdelga represents another important milestone in our commitment to understand and respond to the needs in the Gaucher community, providing more choice for the treatment of patients,” said Genzyme’s President and CEO David Meeker, M.D.
The marketing applications 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.
About Gaucher disease
Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of the enzyme, β-glucosidase (glucocerebrosidase) leading 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. 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. The most common form of Gaucher disease, type 1, generally does not affect the brain.