Myozyme
Alglucosidase alfa
Enzyme replacement therapy drug
Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD).[5] Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease.[2]
It was approved for medical use in the United States in April 2006, as Myozyme[6] and in May 2010, as Lumizyme.[7]