Imiglucerase


Imiglucerase
Imiglucerase
Systematic (IUPAC) name
Human Beta-glucocerebrosidase
Clinical data
AHFS/Drugs.com monograph
MedlinePlus a601149
Licence data EMA:LinkUS FDA:link
Pregnancy cat.  ?
Legal status  ?
Routes Intravenous
Pharmacokinetic data
Half-life 3.6-10.4 min
Identifiers
CAS number 143003-46-7 YesY
ATC code A16AB02
DrugBank BTD00026
UNII Q6U6J48BWY YesY
KEGG D02810 YesY
ChEMBL CHEMBL1201632 N
Chemical data
Formula C2532H3854N672O711S16 
Mol. mass 55597.4 g/mol (unglycosylated)
 N(what is this?)  (verify)

Imiglucerase is a medication used in the treatment of Gaucher's disease.[1][2]

It is a recombinant DNA-produced analogue of human β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation. It is given intravenously after reconstitution as a treatment for Type 1 Gaucher's disease. It is available in formulations containing 200 or 400 units per vial. The specific activity of highly purified human enzyme is 890,000 units/mg.[3] A typical dose is 2.5U/kg every two weeks, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up.[4] It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000.[5] Due to the low profitability (and high expense) of developing medications for rare conditions, imiglucerase has been granted orphan drug status in the USA, Australia, and Japan.[6]

Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2117.[7]

See also

  • Other drugs for the treatment of Gaucher's disease

References

  1. ^ Weinreb NJ (August 2008). "Imiglucerase and its use for the treatment of Gaucher's disease". Expert Opin Pharmacother 9 (11): 1987–2000. doi:10.1517/14656566.9.11.1987. PMID 18627336. http://www.expertopin.com/doi/abs/10.1517/14656566.9.11.1987. 
  2. ^ Starzyk K, Richards S, Yee J, Smith SE, Kingma W (February 2007). "The long-term international safety experience of imiglucerase therapy for Gaucher disease". Mol. Genet. Metab. 90 (2): 157–63. doi:10.1016/j.ymgme.2006.09.003. PMID 17079176. http://linkinghub.elsevier.com/retrieve/pii/S1096-7192(06)00297-6. 
  3. ^ Pentchev et al.; Brady, RO; Blair, HE; Britton, DE; Sorrell, SH (August 1978). "Gaucher disease: Isolation and comparison of normal and mutant glucocerebrosidase from human spleen tissue". Proc. Natl. Acad. Sci. USA 75 (8): 3970–3973. doi:10.1073/pnas.75.8.3970. PMC 392911. PMID 29293. http://www.pubmedcentral.nih.gov/articlerender.fcgi?tool=pmcentrez&artid=392911. 
  4. ^ "Cerezyme® (imiglucerase for injection) Genzyme product data sheet". http://www.cerezyme.com/global/pi.pdf. 
  5. ^ Balancing innovation, access, and profits -- marketing exclusivity for biologics, Alfred B. Engelberg et al., N Engl J Med 361:1917
  6. ^ "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs.". http://www.orpha.net/consor/cgi-bin/Drugs_Search.php?lng=EN&data_id=23&Substance=Imiglucerase&search=Drugs_Search_SubstanceTradename&data_type=Product&diseaseType=Drug&Typ=Sub&title=&diseaseGroup=. 
  7. ^ Erin Ailworth and Robert Weisman (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe. http://www.boston.com/business/healthcare/articles/2009/06/17/genzyme_temporarily_halts_production_on_2_key_drugs/.