Sebelipase alfa explained

Tradename:Kanuma
Dailymedid:Sebelipase_alfa
Pregnancy Au:B1
Pregnancy Au Comment:[1]
Routes Of Administration:Intravenous infusion
Atc Prefix:A16
Atc Suffix:AB14
Legal Au:S4
Legal Au Comment:[2] [3]
Legal Ca:Rx-only
Legal Ca Comment:[4]
Legal Uk:POM
Legal Us:Rx-only
Legal Eu:Rx-only
Legal Status:Rx-only
Elimination Half-Life:0.1 hours
Drugbank:DB11563
Kegg:D10377
Chemspiderid:none

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).[5] [6] It is administered via intraveneous infusion.[7] It was approved for medical use in the European Union and in the United States in 2015.

Medical uses

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

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

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]

Notes and References

  1. Web site: Sebelipase alfa (Kanuma) Use During Pregnancy . Drugs.com . 14 June 2019 . 4 May 2020.
  2. Web site: Prescription medicines: registration of new chemical entities in Australia, 2017 . Therapeutic Goods Administration (TGA) . 21 June 2022 . 9 April 2023.
  3. Web site: Prescription medicines and biologicals: TGA annual summary 2017 . Therapeutic Goods Administration (TGA) . 21 June 2022 . 31 March 2024.
  4. Web site: Genetic disorders . . 9 May 2018 . 13 April 2024.
  5. Web site: Kanuma EPAR . European Medicines Agency (EMA) . 17 September 2018 . 4 May 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  6. Web site: Kanuma (Sebelipase alfa) . U.S. Food and Drug Administration (FDA) . 6 January 2016 . 4 May 2020.
  7. Web site: Kanuma- sebelipase alfa injection, solution, concentrate . DailyMed . 18 December 2018 . 4 May 2020.
  8. News: FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients . FDA . 8 December 2015 . 10 December 2015.
  9. Sheridan C . 7593902 . FDA approves 'farmaceutical' drug from transgenic chickens . Nature Biotechnology . 34 . 2 . 117–9 . February 2016 . 26849497 . 10.1038/nbt0216-117 .
  10. Burton BK, Balwani M, Feillet F, Barić I, Burrow TA, Camarena Grande C, Coker M, Consuelo-Sánchez A, Deegan P, Di Rocco M, Enns GM, Erbe R, Ezgu F, Ficicioglu C, Furuya KN, Kane J, Laukaitis C, Mengel E, Neilan EG, Nightingale S, Peters H, Scarpa M, Schwab KO, Smolka V, Valayannopoulos V, Wood M, Goodman Z, Yang Y, Eckert S, Rojas-Caro S, Quinn AG . 6 . A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency . The New England Journal of Medicine . 373 . 11 . 1010–20 . September 2015 . 26352813 . 10.1056/NEJMoa1501365 . free . 11577/3168673 . free .
  11. Web site: New Drugs Online Report for sebelipase alfa. UK Medicines Information. 10 December 2015. 4 March 2016. https://web.archive.org/web/20160304193606/http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp?newDrugID=5419. dead.

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