Sebelipase alfa

Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the enzyme lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D).[3][4] It is administered via intraveneous infusion.[2] It was approved for medical use in the European Union and in the United States in 2015.[5][3][2][4]

Sebelipase alfa
Clinical data
Trade namesKanuma
AHFS/Drugs.comMonograph
License data
Pregnancy
category
Routes of
administration		Intravenous infusion
ATC code
Legal status
Legal status
  • UK: POM (Prescription only)
  • US: ℞-only [2]
  • EU: Rx-only [3]
  • In general: ℞ (Prescription only)
Pharmacokinetic data
Elimination half-life0.1 hours
Identifiers
DrugBank
ChemSpider
  • none
KEGG

Medical uses

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency.[3]

History

Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production, chickens are genetically modified to produce the recombinant form of LAL (rhLAL) in their egg white. After extraction and purification it becomes available as the medication.[6] On 8 December 2015 the FDA announced that its approval came from two centers: The Center for Drug Evaluation and Research (CDER) approved the human therapeutic application of the medication, while the Center for Veterinary Medicine (CVM) approved the application for a recombinant DNA construct in genetically engineered chicken to produce rhLAL in their egg whites.[7] At the time it gained FDA approval Kanuma was the first only drug manufactured in chicken eggs and intended for use in humans.[6]

Sebelipase alfa is an orphan drug; its effectiveness was published after a phase 3 trial in 2015.[8] The disease of LAL affects < 0.2 in 10,000 people in the EU.[5]

References
  1. "Sebelipase alfa (Kanuma) Use During Pregnancy". Drugs.com. 14 June 2019. Retrieved 4 May 2020.
  2. "Kanuma- sebelipase alfa injection, solution, concentrate". DailyMed. 18 December 2018. Retrieved 4 May 2020.
  3. "Kanuma EPAR". European Medicines Agency (EMA). Retrieved 4 May 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. "Kanuma (Sebelipase alfa)". U.S. Food and Drug Administration (FDA). 6 January 2016. Retrieved 4 May 2020.
  5. "New Drugs Online Report for sebelipase alfa". UK Medicines Information. Archived from the original on 4 March 2016. Retrieved 10 December 2015.
  6. Sheridan C (February 2016). "FDA approves 'farmaceutical' drug from transgenic chickens". Nature Biotechnology. 34 (2): 117–9. doi:10.1038/nbt0216-117. PMID 26849497. S2CID 7593902.
  7. "FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients". FDA. 8 December 2015. Retrieved 10 December 2015.
  8. Burton BK, Balwani M, Feillet F, Barić I, Burrow TA, Camarena Grande C, et al. (September 2015). "A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency". The New England Journal of Medicine. 373 (11): 1010–20. doi:10.1056/NEJMoa1501365. PMID 26352813.


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