Sebelipase alfa
{{Use dmy dates|date=May 2020}}
{{Infobox drug
| IUPAC_name =
| image =
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| pronounce =
| tradename = Kanuma
| Drugs.com = {{Drugs.com|monograph|kanuma}}
| MedlinePlus =
| DailyMedID = Sebelipase_alfa
| pregnancy_AU = B1
| pregnancy_AU_comment = {{cite web | title=Sebelipase alfa (Kanuma) Use During Pregnancy | website=Drugs.com | date=14 June 2019 | url=https://www.drugs.com/pregnancy/sebelipase-alfa.html | access-date=4 May 2020}}
| pregnancy_category=
| routes_of_administration = Intravenous infusion
| ATC_prefix = A16
| ATC_suffix = AB14
| legal_AU = S4
| legal_AU_comment = {{cite web | title=Prescription medicines: registration of new chemical entities in Australia, 2017 | website=Therapeutic Goods Administration (TGA) | date=21 June 2022 | url=https://www.tga.gov.au/resources/publication/publications/prescription-medicines-registration-new-chemical-entities-australia-2017 | access-date=9 April 2023}}{{cite web | title=Prescription medicines and biologicals: TGA annual summary 2017 | website=Therapeutic Goods Administration (TGA) | date=21 June 2022 | url=https://www.tga.gov.au/resources/publication/publications/prescription-medicines-and-biologicals-tga-annual-summary-2017 | access-date=31 March 2024}}
| legal_CA = Rx-only
| legal_CA_comment = {{cite web | title=Genetic disorders | website=Health Canada | date=9 May 2018 | url=https://www.canada.ca/en/services/health/drug-health-products/drug-medical-device-highlights-2017/approved-drugs/genetic-disorders.html | access-date=13 April 2024}}
| legal_DE =
| legal_NZ =
| legal_UK = POM
| legal_US = Rx-only
| legal_EU = Rx-only
| legal_UN =
| legal_status = Rx-only
| bioavailability =
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| elimination_half-life = 0.1 hours
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| CAS_number = 1276027-63-4
| UNII_Ref = {{fdacite|correct|FDA}}
| UNII = K4YTU42T8G
| PubChem =
| DrugBank = DB11563
| KEGG = D10377
| ChemSpiderID = none
| chemical_formula =
| molecular_weight =
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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).{{cite web | title=Kanuma EPAR | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/kanuma | access-date=4 May 2020}} Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.{{cite web | title=Kanuma (Sebelipase alfa) | website=U.S. Food and Drug Administration (FDA) | date=6 January 2016 | url=https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/125561Orig1s000TOC.cfm | access-date=4 May 2020}} It is administered via intraveneous infusion.{{cite web | title=Kanuma- sebelipase alfa injection, solution, concentrate | website=DailyMed | date=18 December 2018 | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=83a77b9d-42f0-4645-8420-786285da7326 | access-date=4 May 2020}} It was approved for medical use in the European Union and in the United States in 2015.
Medical uses
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.{{cite news | url= https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm476013.htm |title= FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients |publisher=FDA |date=8 December 2015 |access-date=10 December 2015}} At the time it gained FDA approval Kanuma was the first only drug manufactured in chicken eggs and intended for use in humans.{{cite journal | vauthors = Sheridan C | s2cid = 7593902 | title = FDA approves 'farmaceutical' drug from transgenic chickens | journal = Nature Biotechnology | volume = 34 | issue = 2 | pages = 117–9 | date = February 2016 | pmid = 26849497 | doi = 10.1038/nbt0216-117 }}
Sebelipase alfa is an orphan drug; its effectiveness was published after a phase 3 trial in 2015.{{cite journal | vauthors = 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 | display-authors = 6 | title = A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency | journal = The New England Journal of Medicine | volume = 373 | issue = 11 | pages = 1010–20 | date = September 2015 | pmid = 26352813 | doi = 10.1056/NEJMoa1501365 | doi-access = free | hdl = 11577/3168673 | hdl-access = free }} The disease of LAL affects < 0.2 in 10,000 people in the EU.{{cite web| url=http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp?newDrugID=5419| title=New Drugs Online Report for sebelipase alfa| publisher=UK Medicines Information| access-date=10 December 2015| archive-date=4 March 2016| archive-url=https://web.archive.org/web/20160304193606/http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp?newDrugID=5419| url-status=dead}}
References
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{{Other alimentary tract and metabolism products}}
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