Imiglucerase

Imiglucerase
Clinical data
Trade namesCerezyme
AHFS/Drugs.comMonograph
MedlinePlusa601149
License data
Routes of
administration
Intravenous infusion
ATC code
Legal status
Legal status
Pharmacokinetic data
Metabolismprobably proteolysis
Elimination half-life3.6–10.4 min
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC2532H3854N672O711S16
Molar mass55597.4 (unglycosylated) g·mol−1
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Imiglucerase is a medication used in the treatment of Gaucher's disease.[1][2]

It is a recombinant DNA-produced analogue of the human enzyme 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 and Type 3[3] 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.[4] 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.[5] It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000.[6] Imiglucerase has been granted orphan drug status in the United States, Australia, and Japan.[7]

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.[8]

Side effects

The most common side effect is hypersensitivity, which occurs in about 3% of patients. It is associated with symptoms such as cough, shortness of breath, rashes, itching, and angiooedema. Less common side effects include dizziness, headache, nausea, diarrhoea, and reactions at the injection site; they are found in less than 1% of patients.[3]

Interactions

No clinical interaction studies have been conducted.[3] Miglustat appears to increase the clearance of imiglucerase by 70%, resulting in decreased enzyme activity.[9]

See also

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.
  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.
  3. ^ a b c Austria-Codex (in German). Vienna: Österreichischer Apothekerverlag. 2018. Cerezyme 400 U-Pulver für ein Konzentrat zur Herstellung einer Infusionslösung.
  4. ^ Pentchev; Brady, RO; Blair, HE; Britton, DE; Sorrell, SH; et al. (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.
  5. ^ "Cerezyme (imiglucerase for injection) Genzyme product data sheet" (PDF). Archived from the original (PDF) on 2003-06-05.
  6. ^ Alfred B. Engelberg, Aaron S. Kesselheim, and Jerry Avorn (November 12, 2009). "Perspective: Balancing Innovation, Access, and Profits — Market Exclusivity for Biologics". N Engl J Med. 361 (20): 1917–1919. doi:10.1056/NEJMp0908496. PMID 19828525.CS1 maint: Uses authors parameter (link)
  7. ^ "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs".
  8. ^ Erin Ailworth; Robert Weisman (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe.
  9. ^ Drug interactions between imiglucerase and miglustat. Accessed 2019-04-11.