Sebelipase alfa

Sebelipase alfa
Names
Trade namesKanuma
Clinical data
Drug classEnzyme[1]
Main usesLysosomal acid lipase deficiency (LAL-D)[2]
Side effectsHeadache, fever, throat pain, nausea, constipation, allergic reactions[1]
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Pregnancy
category
  • AU: B1[3]
  • US: N (Not classified yet)[3]
    Routes of
    use    		Intravenous infusion
    Typical dose1 mg/kg[1]
    External links
    AHFS/Drugs.comMonograph
    Legal
    License data
    Legal status
    • UK: POM (Prescription only)
    • US: ℞-only [4]
    • EU: Rx-only [2]
    • In general: ℞ (Prescription only)
    Pharmacokinetics
    Elimination half-life0.1 hours

    Sebelipase alfa, sold under the brand name Kanuma, is a medication used to treat lysosomal acid lipase deficiency (LAL-D).[2] Benefit is clearest in Wolman disease.[1] It is given by injection into a vein.[4]

    Common side effects include headache, fever, throat pain, nausea, and constipation.[1] Other side effects may include allergic reactions.[1] It is a recombinant form of the lysosomal acid lipase (LAL) enzyme, which helps to break down lipids.[1][2]

    Sebelipase alfa was approved for medical use in Europe and the United States in 2015.[2][1] In Canada it cost about 892,000 CAD for an adult per year in 2018.[5] In the United States this amount costs about 970,000 USD per year as of 2021.[6] Its use was rejected by the United Kingdom in 2017.[7]

    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]

    Dosage

    It is given at a dose of 1 mg/kg every two weeks.[1]

    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.[8] 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.[9] At the time it gained FDA approval Kanuma was the first only drug manufactured in chicken eggs and intended for use in humans.[8]

    Sebelipase alfa is an orphan drug; its effectiveness was published after a phase 3 trial in 2015.[10] The disease of LAL affects < 0.2 in 10,000 people in the EU.[11]

    Society and culture

    Economics

    According to an estimate by a Barclays analyst in 2015, the drug was expected to be priced at about US$375,000 per year.[11]

    References

    1. 1 2 3 4 5 6 7 8 9 "Sebelipase Alfa Monograph for Professionals". Drugs.com. Retrieved 11 October 2021.
    2. 1 2 3 4 5 6 "Kanuma EPAR". European Medicines Agency (EMA). Archived from the original on 27 October 2020. Retrieved 4 May 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
    3. 1 2 "Sebelipase alfa (Kanuma) Use During Pregnancy". Drugs.com. 14 June 2019. Archived from the original on 25 January 2021. Retrieved 4 May 2020.
    4. 1 2 "Kanuma- sebelipase alfa injection, solution, concentrate". DailyMed. 18 December 2018. Archived from the original on 2 July 2019. Retrieved 4 May 2020.
    5. ↑ "Pharmacoeconomic Review Report: Sebelipase Alfa (Kanuma): (Alexion Pharmaceuticals, Inc.): Indication: Indicated for the treatment of infants, children, and adults diagnosed with lysosomal acid lipase (LAL) deficiency". Canadian Agency for Drugs and Technologies in Health. 2018. PMID 30920785. Archived from the original on 12 October 2021. Retrieved 11 October 2021. {{cite journal}}: Cite journal requires |journal= (help)
    6. ↑ "Kanuma Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 28 January 2021. Retrieved 11 October 2021.
    7. ↑ "Sebelipase alfa for treating lysosomal acid lipase deficiency". NICE. Archived from the original on 20 February 2017. Retrieved 11 October 2021.
    8. 1 2 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.
    9. ↑ "FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients". FDA. 8 December 2015. Archived from the original on 25 January 2018. Retrieved 10 December 2015.
    10. ↑ 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.
    11. 1 2 "New Drugs Online Report for sebelipase alfa". UK Medicines Information. Archived from the original on 4 March 2016. Retrieved 10 December 2015.
    Identifiers:
    • "Sebelipase alfa". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on 29 August 2021. Retrieved 2 December 2020.
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