Imiglucerase

Imiglucerase is a medication used in the treatment of Gaucher's disease.^[2][3]

Imiglucerase

Clinical data
Trade names	Cerezyme
AHFS/Drugs.com	Monograph
MedlinePlus	a601149
License data	EU EMA: by INN US DailyMed: Imiglucerase
Routes of administration	Intravenous infusion
ATC code	A16AB02 (WHO)
Legal status
Legal status	US: ℞-only EU: Rx-only[1]
Pharmacokinetic data
Metabolism	probably proteolysis
Elimination half-life	3.6–10.4 min
Identifiers
IUPAC name Human beta-glucocerebrosidase
CAS Number	154248-97-2 N
DrugBank	DB00053 Y
ChemSpider	none
UNII	Q6U6J48BWY
KEGG	D02810 Y
ChEMBL	ChEMBL1201632 N
Chemical and physical data
Formula	C2532H3854N672O711S16
Molar mass	55597.4 g·mol−1 (unglycosylated)
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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^[4] 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,^[5] meanwhile the enzyme activity produced by recombinant DNA technology is approximately 40 units/mg.^[6] 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.^[7] It is one of more expensive medications, with an annual cost of $200,000 per person in the United States.^[8] Imiglucerase has been granted orphan drug status in the United States, Australia, and Japan.^[9]

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.^[10]

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, diarrhea, and reactions at the injection site; they are found in less than 1% of patients.^[4]

Interactions

No clinical interaction studies have been conducted.^[4] Miglustat appears to increase the clearance of imiglucerase by 70%, resulting in decreased enzyme activity.^[11]

See also

• Other drugs for the treatment of Gaucher's disease
  • Afegostat (development terminated)
  • Eliglustat
  • Miglustat
  • Velaglucerase alfa
  • taliglucerase alfa

References

1. "Cerezyme EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 18 January 2021.
2. Weinreb NJ (August 2008). "Imiglucerase and its use for the treatment of Gaucher's disease". Expert Opinion on Pharmacotherapy. 9 (11): 1987–2000. doi:10.1517/14656566.9.11.1987. PMID 18627336. S2CID 72183308.
3. Starzyk K, Richards S, Yee J, Smith SE, Kingma W (February 2007). "The long-term international safety experience of imiglucerase therapy for Gaucher disease". Molecular Genetics and Metabolism. 90 (2): 157–163. doi:10.1016/j.ymgme.2006.09.003. PMID 17079176.
4. Austria-Codex (in German). Vienna: Österreichischer Apothekerverlag. 2018. Cerezyme 400 U-Pulver für ein Konzentrat zur Herstellung einer Infusionslösung.
5. Pentchev PG, Brady RO, Blair HE, Britton DE, Sorrell SH (August 1978). "Gaucher disease: isolation and comparison of normal and mutant glucocerebrosidase from human spleen tissue". Proceedings of the National Academy of Sciences of the United States of America. 75 (8): 3970–3973. Bibcode:1978PNAS...75.3970P. doi:10.1073/pnas.75.8.3970. PMC 392911. PMID 29293.
6. "Cerezyme Powder for concentrate for solution for infusion, Annex I: Summary of Product Characteristics" (PDF). Sanofi-Aventis Latvia SIA. Retrieved 26 April 2022 – via Ec.europa.eu.
7. "Cerezyme (imiglucerase for injection)" (PDF). Genzyme product data sheet. Archived from the original (PDF) on 2003-06-05.
8. Engelberg AB, Kesselheim AS, Avorn J (November 2009). "Balancing innovation, access, and profits--market exclusivity for biologics". The New England Journal of Medicine. 361 (20): 1917–1919. doi:10.1056/NEJMp0908496. PMID 19828525.
9. "Imiglucerase". Orphanet. Retrieved 26 April 2022.
10. Ailworth E, Weisman R (June 17, 2009). "Virus shuts Genzyme plant, holds up drugs for 8,000". The Boston Globe.
11. "Drug interactions between imiglucerase and miglustat". Drugs.com. Retrieved 11 April 2019.