Alglucosidase alfa

Alglucosidase alfa, sold under the brand name Myozyme among others, 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]

Alglucosidase alfa
Clinical data
Trade namesMyozyme, Lumizyme, others
AHFS/Drugs.comMonograph
License data
Routes of
administration		Intravenous[1]
ATC code
Legal status
Legal status
  • US: FDA approved for children[1]
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC4758H7262N1274O1369S35[2]
Molar mass105338 g·mol−1[2]
 NY (what is this?)  (verify)

Medical uses

Alglucosidase alfa is used as ERT treatment for children.[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 8 years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) is being eliminated.[4]

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.[1]

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 (US$300,000 per year for life).[5]

In 2015, Lumizyme was ranked the costliest drug per patient, with an average charge of $630,159.[6]

References
  1. "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. Retrieved 2008-07-07.
  2. 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. 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=https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407563.htm
  5. Geeta Anand (2007-09-18). "As Costs Rise, New Medicines Face Pushback". Wall Street Journal. Dow Jones & Company. Retrieved 2008-07-07.
  6. https://www1.magellanrx.com/media/604882/2016mrxtrendreport_final.pdf
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