Imiglucerase

Imiglucerase
Names
Trade namesCerezyme
IUPAC name
  • Human beta-glucocerebrosidase
Clinical data
Drug classEnzyme
Main usesGaucher's disease[1]
Side effectsShortness of breath, cough, headache, itchiness, rash[2]
WHO AWaReUnlinkedWikibase error: ⧼unlinkedwikibase-error-statements-entity-not-set⧽
Routes of
use		Intravenous infusion
Typical dose15 to 60 units/kg q 2 wks[1]
External links
AHFS/Drugs.comMonograph
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)

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 2 3 4 5 6 7 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1113. ISBN 978-0857114105.
  2. 1 2 3 4 5 "Imiglucerase Monograph for Professionals". Drugs.com. Archived from the original on 30 March 2016. Retrieved 25 November 2021.
  3. ↑ "Cerezyme EPAR". European Medicines Agency (EMA). Archived from the original on 28 January 2021. Retrieved 18 January 2021.
  4. 1 2 3 "Cerezyme". Archived from the original on 28 January 2021. 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. 1 2 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 Archived 2020-09-29 at the Wayback Machine. Accessed 2019-04-11.
  10. ↑ "Imiglucerase on Orpha.net: The portal for rare diseases and orphan drugs". Archived from the original on 2012-12-29. Retrieved 2021-09-26.
  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.{{cite journal}}: 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. Archived from the original on 22 September 2021. 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. Archived from the original on March 4, 2016. Retrieved September 26, 2021.
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