Imiglucerase

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Imiglucerase
Names
Trade namesCerezyme
Clinical data
Drug classEnzyme
Main usesGaucher's disease[1]
Side effectsShortness of breath, cough, headache, itchiness, rash[2]
Routes of
use
Intravenous infusion
Typical dose15 to 60 units/kg q 2 wks[1]
External links
AHFS/Drugs.comMonograph
US NLMImiglucerase
MedlinePlusa601149
Legal
License data
Legal status
Pharmacokinetics
Metabolismprobably proteolysis
Elimination half-life3.6–10.4 min
Chemical and physical data
FormulaC2532H3854N672O711S16
Molar mass55597.4 g·mol−1 (unglycosylated)
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Imiglucerase, sold under the brand name Cerezyme, is a medication used to treat Gaucher's disease.[1] Specifically it is used for type I and III disease.[1] It is given by injection into a vein.[1] It is used long term.[4]

Common side effects include shortness of breath, cough, headache, itchiness, and rash.[2] Other side effects may include pulmonary hypertension, pneumonia, and allergic reactions.[2] It is a form of the enzyme β-glucocerebrosidase produced by recombinant DNA technology.[2]

Imiglucerase was approved for medical use in the United States in 1994 and Europe in 1997.[2][4] In the United Kingdom 400 units costs the NHS about £1,100 as of 2021.[1] In the United States this amount costs about 1,650 USD.[5]

Medical uses

It is used to improve low red blood cells, low platelets, bone disease, and an enlarged liver or spleen in type I or III Gaucher disease.[4]

Dosage

It is generally started at a dose of 60 units/kg every two weeks, which may be decreased down to 15 units/kg.[1]

It is available in formulations containing 200 or 400 units per vial. It has an activity of 890,000 units/mg.[6] A typical dose is 2.5U/kg three times per week, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up.[7]

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.[8]

Interactions

No clinical interaction studies have been conducted.[8] Miglustat appears to increase the clearance of imiglucerase by 70%, resulting in decreased enzyme activity.[9]

History

Imiglucerase has been granted orphan drug status in the United States, Australia, and Japan.[10]

Society and culture

It is one of more expensive medications, with an annual cost of $200,000 per person in the United States.[11]

In Canada a year of medication costs about 385,000 to 705,000 CAD for a 70 kg person per year.[12]

Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2017.[13]

References

  1. 1.0 1.1 1.2 1.3 1.4 1.5 1.6 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1113. ISBN 978-0857114105.
  2. 2.0 2.1 2.2 2.3 2.4 "Imiglucerase Monograph for Professionals". Drugs.com. Retrieved 25 November 2021.
  3. "Cerezyme EPAR". European Medicines Agency (EMA). Retrieved 18 January 2021.
  4. 4.0 4.1 4.2 "Cerezyme". Retrieved 25 November 2021.
  5. "Imiglucerase Prices, Coupons & Savings Tips - GoodRx". GoodRx. Retrieved 25 November 2021.
  6. 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. Bibcode:1978PNAS...75.3970P. doi:10.1073/pnas.75.8.3970. PMC 392911. PMID 29293.
  7. "Cerezyme (imiglucerase for injection) Genzyme product data sheet" (PDF). Archived from the original (PDF) on 2003-06-05.
  8. 8.0 8.1 Austria-Codex (in Deutsch). Vienna: Österreichischer Apothekerverlag. 2018. Cerezyme 400 U-Pulver für ein Konzentrat zur Herstellung einer Infusionslösung.
  9. Drug interactions between imiglucerase and miglustat. Accessed 2019-04-11.
  10. "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs".
  11. 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)
  12. Information, National Center for Biotechnology; Pike, U. S. National Library of Medicine 8600 Rockville; MD, Bethesda (August 2017). "Table 12, CDR Cost Comparison Table for Drug Therapies for GD1". www.ncbi.nlm.nih.gov. Retrieved 25 November 2021.
  13. Erin Ailworth; Robert Weisman (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe.

External links

Identifiers: