|Trade names||Myozyme, Lumizyme, others|
|Main uses||Pompe disease|
|Side effects||Allergic reactions, fever, headache, sweating, nausea, tiredness, muscle pain|
|Typical dose||20 mg/kg|
|US NLM||Alglucosidase alfa|
|Chemical and physical data|
|Molar mass||105338 g·mol−1|
Common side effects include allergic reactions, fever, headache, sweating, nausea, tiredness, and muscle pain. Other side effects may include anaphylaxis. It is an enzyme that replaces the missing alpha-glucosidase.
Alglucosidase alfa was approved for medical use in the United States and Europe in 2006. In the United Kingdom 50 mg costs the NHS about £360 as of 2021. This amount in the United States is about 900 USD.
Alglucosidase alfa is indicated for people with Pompe disease (GAA deficiency).
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 eight years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) is being eliminated.
It is given at a dose of 20 mg/kg every two weeks.
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.
It is the first drug available to treat this disease.
In 2015, Lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.
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