Alglucosidase alfa

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).

[6] Chemically, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase.

[9] Common observed adverse reactions to alglucosidase alfa treatment are pneumonia, respiratory complications, infections and fever.

More serious reactions reported include heart and lung failure and allergic shock.

[10] In 2015, alglucosidase alfa was ranked the costliest drug per patient, with an average charge of US$630,159.