Alglucosidase alfa

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Alglucosidase alfa (Lumizyme, Myozyme, Genzyme) is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD).[1] 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.[2]

Status

Orphan drug pharmaceutical company, Genzyme, markets alglucosidase alfa as "Myozyme". In 2006, the U.S. Food and Drug Administration (FDA) approved Myozyme as a suitable ERT treatment for children.[2] Some health plans have refused to subsidize Myozyme for adult patients because it lacks approval for treatment in adults, as well as its high cost (US$300,000/yr for life).[3]

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.[2]

References

  1. Kishnani PS; Corzo D; Nicolino M; et al. (2007). "Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease". Neurology. 68 (2): 99–109. doi:10.1212/01.wnl.0000251268.41188.04. PMID 17151339. Unknown parameter |author-separator= ignored (help)
  2. 2.0 2.1 2.2 "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. Retrieved 2008-07-07.
  3. Geeta Anand (2007-09-18). "As Costs Rise, New Medicines Face Pushback". Wall Street Journal. Dow Jones & Company. Retrieved 2008-07-07.
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