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).[2][3] It is administered via intraveneous infusion.[4] It was approved for medical use in the European Union and in the United States in 2015.[5][2][4][3]

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
    • 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.[2]

    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 December 8, 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]

    According to an estimate by a Barclays analyst in 2015, the drug was expected to be priced at about US$375,000 per year.[5]

    References
    1. "Sebelipase alfa (Kanuma) Use During Pregnancy". Drugs.com. 14 June 2019. Retrieved 4 May 2020.
    2. "Kanuma EPAR". European Medicines Agency (EMA). Retrieved 4 May 2020. This article incorporates text from this source, which is in the public domain.
    3. "Kanuma (Sebelipase alfa)". U.S. Food and Drug Administration (FDA). 6 January 2016. Retrieved 4 May 2020.
    4. "Kanuma- sebelipase alfa injection, solution, concentrate". DailyMed. 18 December 2018. Retrieved 4 May 2020.
    5. "New Drugs Online Report for sebelipase alfa". UK Medicines Information. Retrieved 10 December 2015.
    6. Sheridan, Cormac (5 February 2016). "FDA approves 'farmaceutical' drug from transgenic chickens". Nature Biotechnology. 34 (2): 117–119. doi:10.1038/nbt0216-117. PMID 26849497.
    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, B. K.; et al. (10 September 2015). "A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency". New England Journal of Medicine. 373 (11): 1010–1020. doi:10.1056/NEJMoa1501365. PMID 26352813.


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