FDAnews Drug Daily Bulletin

FDA APPROVES FIRST TREATMENT FOR POMPE DISEASE

May 9, 2006
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The FDA has approved a biologics license application (BLA) for Myozyme (alglucosidase alfa, rhGAA) as the first treatment for Pompe disease.

This rare, inherited condition is caused by a lack of the enzyme acid alpha-glucosidase, which results in a dramatic reduction in the patient's muscle and respiratory function. Myozyme, manufactured by Genzyme, also already been designated an orphan drug.