Genzyme Submits BLA for Myozyme for Treatment of Pompe Disease

Category: Archive
July 28, 2005

Pivotal Trial Meets Primary Endpoint

Genzyme Corp. (Nasdaq: GENZ) announced today that it has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration for Myozyme® (alglucosidase alfa). If approved, Myozyme would be the first treatment developed for patients with Pompe disease, a debilitating and often fatal muscle disorder resulting from an inherited enzyme deficiency. The Myozyme BLA is expected to receive Priority Review by the FDA, which means the agency would be required to act on the application within six months.

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