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| Trade names | Myozyme, Lumizyme, others |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | Intravenous[1][2] |
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| Chemical and physical data | |
| Formula | C4758H7262N1274O1369S35[5] |
| Molar mass | 105338 g·mol−1[5] |
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Alglucosidase alfa, sold under the brand nameMyozyme among others, is anenzyme replacement therapy (ERT)orphan drug for treatment of Pompe disease (Glycogen storage disease type II), ararelysosomal storage disorder (LSD).[6]Chemically, the drug is ananalog 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[7] and in May 2010, as Lumizyme.[8]
Alglucosidase alfa is indicated for people with Pompe disease (GAA deficiency).[1]
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, theRisk Evaluation and Mitigation Strategy (REMS) is being eliminated.[9]
Common observed adverse reactions to alglucosidase alfa treatment arepneumonia, respiratory complications,infections andfever. More serious reactions reported includeheart andlung failure andallergic shock. Myozyme boxes carry warnings regarding the possibility of life-threatening allergic response.[2]
Some American health plans have refused to subsidize alglucosidase alfa for adults because it lacks approval for treatment in adults, as well as its high cost (US$ 300,000 per year for life).[10]
In 2015, alglucosidase alfa was ranked the costliest drug per patient, with an average charge of US$630,159.[11]
