New First-Line Oral Therapy for Gaucher Disease Approved

Genzyme announced that the Food and Drug Administration (FDA) has approved Cerdelga (eliglustat) capsules for the treatment of certain adult Gaucher disease type 1 patients. Cerdelga is the only first-line oral therapy for this population.

Cerdelga is a is a specific inhibitor of glucosylceramide synthase and acts as a substrate reduction therapy for GD1. In clinical trials, Cerdelga reduced spleen and liver size, and improved anemia and thrombocytopenia.

The approval was based on efficacy data from two Phase 3 studies for Cerdelga: one in patients new to therapy (Trial 1), and the other in patients switching from approved enzyme replacement therapies (Trial 2). The submission also included 4 years of efficacy data from the Cerdelga Phase 2 study.

Cerdelga will be available in 84mg strength capsules in 1- and 4-pack cartons with each pack containing 14 capsules. Cerdelga will be available within one month.

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