Almost a year after its application, the Food and Drug Administration granted Genzyme marketing approval for Myozyme® (alglucosidase alfa) in the United States. Myozyme has been approved for the treatment of patients with Pompe disease, a debilitating, progressive and often fatal muscle disorder This genetic disease is caused by a Deficiency in the enzyme acid alpha-glucosidase (GAA), leading to an accumulation of glycogen, subsequently causing progressive muscle weakness throughout the body, particularly in the heart, skeletal muscles, liver, and nervous system.

Myozyme has received orphan drug designation in the United States, which provides seven years of market exclusivity. The orphan drug law is designed to encourage the development of treatments for rare disorders such as Pompe disease, for which no therapies have existed previously. Genzyme expects to launch Myozyme in the United States within two weeks.

The product, also approved in the EU, is the first treatment ever approved for Pompe disease and the first for a genetic neuromuscular disorder.

[Source: Genzyme]