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.