Alglucosidase alfa

Alglucosidase alfa
Systematic (IUPAC) name
Human glucosidase, prepro-α-[199-arginine,223-histidine] [1]
Clinical data
AHFS/Drugs.com monograph
Pregnancy cat.  ?
Legal status FDA approved for children[2]
Routes Intravenous[2]
Identifiers
CAS number 420794-05-0 N
ATC code A16AB07
DrugBank DB01272
UNII DTI67O9503 N
KEGG D03207 N
Chemical data
Formula C4758H7262N1274O1369S35 [1] 
Mol. mass 105338 [1]
 N(what is this?)  (verify)

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.[2]

Contents

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).[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.[2]

References

  1. ^ a b c American Medical Association ( USAN). "Alglucosidase alfa" (Microsoft Word). STATEMENT ON A NONPROPRIETARY NAME ADOPTED BY THE USAN COUNCIL. http://www.ama-assn.org/ama1/pub/upload/mm/365/alglucosidase_alfa.doc. Retrieved 2008. 
  2. ^ a b c d e "FDA Approves First Treatment for Pompe Disease" (Press release). FDA. 2006-04-28. http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html. Retrieved 2008-07-07. 
  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). http://online.wsj.com/article/SB119007210553130427.html. Retrieved 2008-07-07. 

External links

Other alimentary tract and metabolism products (A16)
Amino acids and derivatives
Enzymes
Various alimentary tract
and metabolism products

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