Alglucosidase alfa

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Alglucosidase alfa
Clinical data
License data
Routes of
ATC code
Legal status
Legal status
  • US: FDA approved for children[1]
CAS Number
  • none
Chemical and physical data
Molar mass105,338 g·mol−1[2]
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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).[3] 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.[1]


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.[1] 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).[4]

On August 1, 2014 the U.S. Food and Drug Administration announced the approval of Lumizyme (alglucosidase alfa) for treatment of patients with infantile-onset Pompe disease, including patients who are less than 8 years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) known as the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program is being eliminated.[5]

For 2016, Lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.[6]

Side effects[edit]

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.[1]


  1. ^ a b c d e "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. Retrieved 2008-07-07.
  2. ^ a b c American Medical Association (USAN). "Alglucosidase alfa" (Microsoft Word). Statement on a Nonproprietary Name Adopted by the USAN Council. Retrieved 18 December 2007.
  3. ^ 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.
  4. ^ Geeta Anand (2007-09-18). "As Costs Rise, New Medicines Face Pushback". Wall Street Journal. Dow Jones & Company. Retrieved 2008-07-07.
  5. ^ cite press release |title=FDA expands approval of drug to treat Pompe disease to patients of all ages; removes risk mitigation strategy requirements |publisher=FDA |date=2014-08-14 |url=
  6. ^

External links[edit]