Alglucosidase alfa


Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy orphan drug for treatment of Pompe disease, a rare lysosomal storage disorder.
Chemically speaking, 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.

Medical uses

Alglucosidase alfa is used as ERT treatment for children.
In 2014 the U.S. Food and Drug Administration announced the approval of alglucosidase alfa for treatment of people with infantile-onset Pompe disease, including people who are less than 8 years of age. In addition, the Risk Evaluation and Mitigation Strategy is being eliminated.

Side effects

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. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.

Cost

Some health plans have refused to subsidize Myozyme for adults because it lacks approval for treatment in adults, as well as its high cost.
In 2015, Lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.