New First-Line Oral Therapy for Gaucher Disease Approved

 CERDELGA (eliglustat) capsules by Genzyme
CERDELGA (eliglustat) capsules by Genzyme

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.

RELATED: Cerdelga NDA Granted Priority Review for Gaucher Disease

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.

For more information call (800) 745-4447 or visit