Alglucosidase alfa


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


Wikimedia Foundation. 2010.

Look at other dictionaries:

  • alglucosidase alfa — al·glu·co·si·dase al·fa (al gloo koґsĭ dās alґfə) a recombinant form of the enzyme α 1,4 glucosidase, used in treatment of glycogen storage disease, type II; administered by intravenous infusion …   Medical dictionary

  • АТХ код A16 — АТХ A «Препараты, влияющие на пищеварительный тракт и обмен веществ» АТХ код A01 Стоматологические препараты АТХ код A02 Препараты для лечения заболеваний, связанных с нарушением кислотности АТХ код A03 Препараты для лечения функциональных… …   Википедия

  • Morbus Pompe — Klassifikation nach ICD 10 E74.0 Glykogenspeicherkrankheit …   Deutsch Wikipedia

  • Enfermedad de Pompe — Clasificación y recursos externos CIE 10 E …   Wikipedia Español

  • Glycogen storage disease type II — Infobox Disease Name = Glycogen storage disease type II Caption = DiseasesDB = 5296 ICD10 = ICD10|E|74|0|e|70 ICD9 = ICD9|271.0 ICDO = OMIM = 232300 MedlinePlus = eMedicineSubj = med eMedicineTopic = 908 eMedicine mult = eMedicine2|ped|1866 |… …   Wikipedia

  • Maladie de Pompe — Référence MIM 232300 Transmission Récessive Chromosome 17 q25.2 q25.3 Gène GAA Empreinte parentale Non …   Wikipédia en Français

  • Carnitine — Systematic (IUPAC) name 3 hydroxy 4 (trimethylazaniumyl)butanoate Clinical data AHFS/Drugs.com …   Wikipedia

  • Tetrahydrobiopterin — Systematic (IUPAC) name (6R) 2 Amino 6 [(1R,2S) 1,2 dihydroxypropyl] 5,6,7,8 tetrahydropteridin 4(1H) one Clinical data …   Wikipedia

  • Alglucerase — Systematic (IUPAC) name Human beta glucocerebrosidase Clinical data AHFS/Drugs.com monograph MedlinePlus …   Wikipedia

  • Imiglucerase — Systematic (IUPAC) name Human Beta glucocerebrosidase Clinical data AHFS/Drugs.com monograph MedlinePlus …   Wikipedia