FDA approves Genzyme's Myozyme for all patients with Pompe disease

By Lisa Kerner

Erie, Pa., April 28 - The Food and Drug Administration granted U.S. marketing approval of Genzyme Corp.'s Myozyme (alglucosidase alfa) for the treatment of all patients with Pompe disease, a debilitating, progressive and often fatal disorder affecting fewer than 10,000 people worldwide.

Myozyme already has orphan drug designation in the United States, providing it with seven years of market exclusivity.

The product is the first treatment approved for Pompe disease and the first for an inherited muscle disorder, according to a company news release.

Genzyme said the Myozyme label indicates that the product has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease, but that it has not been adequately studied in patients with other forms of Pompe disease.

The label also carries a boxed warning with information on the potential risk of hypersensitivity reactions associated with use of the product.

Genzyme recently completed enrollment in its placebo-controlled study of 90 patients with late-onset Pompe disease.

The company currently manufactures Myozyme in the United States but plans to produce Myozyme at its new protein manufacturing facility in Geel, Belgium, and its new fill/finish facility in Waterford, Ireland.

Genzyme is a biotechnology company based in Cambridge, Mass.

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