burosumab
{{Short description|Monoclonal antibody designed to treat X-linked hypophosphatemia}}
{{Use dmy dates|date=February 2020}}
{{Infobox drug
| drug_name =
| INN =
| type = mab
| image =
| width =
| alt =
| caption =
| mab_type = mab
| source = u
| target = FGF 23
| pronounce = bur OH sue mab
| tradename = Crysvita
| Drugs.com = {{drugs.com|monograph|burosumab-twza}}
| MedlinePlus = a618034
| licence_CA =
| licence_EU = yes
| DailyMedID = Burosumab
| licence_US =
| pregnancy_AU = B3
| pregnancy_AU_comment = {{cite web | title=Crysvita | website=Therapeutic Goods Administration (TGA) | date=17 September 2021 | url=https://www.tga.gov.au/apm-summary/crysvita | access-date=17 September 2021}}{{cite web | title=Updates to the Prescribing Medicines in Pregnancy database | website=Therapeutic Goods Administration (TGA) | date=12 May 2022 | url=https://www.tga.gov.au/resources/resource/guidance/updates-prescribing-medicines-pregnancy-database | access-date=13 May 2022}}
| pregnancy_category=
| routes_of_administration = Subcutaneous
| class =
| ATC_prefix = M05
| ATC_suffix = BX05
| ATC_supplemental =
| legal_AU = S4
| legal_AU_comment = {{cite web | title=Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial | website=Therapeutic Goods Administration (TGA) | url=https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 | access-date=17 September 2021 | archive-date=17 September 2021 | archive-url=https://web.archive.org/web/20210917193625/https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 | url-status=dead }}{{cite web | title=Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial | website=Therapeutic Goods Administration (TGA) | url=https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 | format=PDF | access-date=17 September 2021 | archive-date=17 September 2021 | archive-url=https://web.archive.org/web/20210917193625/https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 | url-status=dead }}
| legal_BR =
| legal_BR_comment =
| legal_CA = Rx-only
| legal_CA_comment = / Schedule D{{cite web | title=Crysvita Product information | website=Health Canada | date=25 April 2012 | url=https://health-products.canada.ca/dpd-bdpp/info.do?lang=en&code=97399 | access-date=29 May 2022}}{{cite web | title=Summary Basis of Decision (SBD) for Crysvita | website=Health Canada | date=23 October 2014 | url=https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00429&lang=en | access-date=29 May 2022}}
| legal_DE =
| legal_DE_comment =
| legal_NZ =
| legal_NZ_comment =
| legal_UK = POM
| legal_US = Rx-only
| legal_US_comment = {{cite web | title=Crysvita- burosumab injection | website=DailyMed | url=https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6f5f3556-3197-477a-993c-96b2c29a91ac | access-date=17 September 2021}}{{cite web | title=Drug Approval Package: Crysvita (burosumab-twza) | website=U.S. Food and Drug Administration (FDA) | date=15 May 2018 | url=https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/761068Orig1s000TOC.cfm | archive-url=https://web.archive.org/web/20200228064619/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/761068Orig1s000TOC.cfm | url-status=dead | archive-date=28 February 2020 | access-date=28 February 2020}}
| legal_EU = Rx-only
| legal_EU_comment =
| legal_UN =
| legal_UN_comment =
| legal_status = Rx-only
| bioavailability =
| protein_bound =
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| CAS_number_Ref =
| CAS_number = 1610833-03-8
| CAS_supplemental =
| PubChem =
| IUPHAR_ligand =
| DrugBank_Ref =
| DrugBank = DB14012
| ChemSpiderID_Ref =
| ChemSpiderID = none
| UNII_Ref = {{fdacite|correct|FDA}}
| UNII = G9WJT6RD29
| KEGG_Ref =
| KEGG = D10913
| ChEBI_Ref =
| ChEBI =
| ChEMBL_Ref =
| ChEMBL =
| NIAID_ChemDB =
| PDB_ligand =
| synonyms = KRN-23, KRN23, burosumab-twza
| IUPAC_name =
| C = 6388 | H = 9904 | N = 1700 | O = 2006 | S = 46
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Burosumab, sold under the brand name Crysvita, is a human monoclonal antibody medication approved 2018 for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia.{{cite journal | vauthors=((World Health Organization)) | year=2017 | title=International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77 | journal=WHO Drug Information | volume=31 | issue=1 | page= | hdl=10665/330984 | hdl-access=free }}{{cite web|url= http://www.c-hotline.net/docs/html/KYOW7605/dl/kyow171027_1.pdf|title= Burosumab (KRN23) for X-Linked Hypophosphatemia (XLH)|author= |date= n.d.|access-date= 2018-04-18|archive-url= https://web.archive.org/web/20180418230050/http://www.c-hotline.net/docs/html/KYOW7605/dl/kyow171027_1.pdf|archive-date= 2018-04-18|url-status= dead}}
Medical uses
In the European Union and the United States, burosumab is indicated for the treatment of adults and children ages one year and older with X-linked hypophosphatemia (XLH), a rare, inherited form of rickets. caused by overproduction of a hormone called FGF23 (fibroblast growth factor 23) in bone cells. FGF23 is responsible for blocking phosphate re-absorption in the kidney and the suppression of the vitamin D dependent phosphate absorption in the intestine. Due to the excess activity of FGF23, phosphate levels in the blood are abnormally low (hypophosphatemia), which affects the constitution of bone.{{cite web | title = What Is X-Linked Hypophosphatemia? | url = http://www.ultragenyx.com/patients/xlh/ | publisher = Ultragenyx Pharmaceutical | access-date = 22 February 2020 | archive-date = 23 June 2021 | archive-url = https://web.archive.org/web/20210623201520/https://www.ultragenyx.com/patients/xlh/ | url-status = dead }}
In the United States, burosumab is also approved to treat people age two and older with tumor-induced osteomalacia (TIO), a rare disease which is characterized by the development of tumors causing weakened and softened bones.{{cite press release | title=FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening | website=U.S. Food and Drug Administration | date=18 June 2020 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-disease-causes-low-phosphate-blood-levels-bone-softening | archive-url=https://web.archive.org/web/20200618215939/https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-disease-causes-low-phosphate-blood-levels-bone-softening | url-status=dead | archive-date=18 June 2020 | access-date=19 June 2020}} {{PD-notice}} The tumors associated with TIO release fibroblast growth factor 23 (FGF23) which lowers phosphate levels.
Adverse effects
In trials, injection site reactions were very common, occurring in 52–58% of patients; they were generally mild in severity, and resolved on their own in 1–3 days.{{Drugs.com|pro|crysvita}}
Legal status
It was approved for use in the European Union in February 2018 to treat children one year of age and older and adolescents with growing skeletons who have X-linked hypophosphataemia with radiographic evidence of bone disease .{{cite web | title=Crysvita EPAR | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/crysvita | access-date=1 March 2020}} {{PD-notice}}
In April 2018, the U.S. Food and Drug Administration (FDA) approved burosumab for its intended purpose in patients aged one year and older.{{Cite press release|title=FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia|date=17 April 2018|publisher=U.S. Food and Drug Administration (FDA)|url=https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-inherited-form-rickets-x-linked-hypophosphatemia|archive-url=https://web.archive.org/web/20191211193233/https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-inherited-form-rickets-x-linked-hypophosphatemia|url-status=dead|archive-date=11 December 2019}} {{PD-notice}} The FDA approval fell under both the breakthrough therapy and orphan drug designations.{{cite web | title=Crysvita Orphan Drug Designation | website=U.S. Food and Drug Administration (FDA) | date=24 December 1999 | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=296109 | archive-url=https://web.archive.org/web/20211104155919/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=296109 | url-status=dead | archive-date=4 November 2021 | access-date=27 February 2020}}
The FDA considered it to be a first-in-class medication.{{cite report | title=New Drug Therapy Approvals 2018 | website=U.S. Food and Drug Administration (FDA) | date=January 2019 | url=https://www.fda.gov/media/120357/download | archive-url=https://web.archive.org/web/20190826114431/https://www.fda.gov/media/120357/download | url-status=dead | archive-date=26 August 2019 | format=PDF | access-date=16 September 2020}}
In 2018, the National Institute for Health and Care Excellence in England and Wales raised concerns regarding the incremental cost-effectiveness of the new treatment{{cite news|title=U.K. cost watchdogs turn away rare disease med Crysvita|newspaper=Fierce Pharma |date=15 June 2018|url=https://www.fiercepharma.com/pharma/u-k-cost-watchdogs-turn-away-rare-disease-med-crysvita}} but as of 2019 the drug was available through a simple discount scheme.{{Cite web|url=https://www.nice.org.uk/guidance/hst8/chapter/1-Recommendations|title=1 Recommendations {{!}} Burosumab for treating X-linked hypophosphataemia in children and young people. Guidance NICE|website=www.nice.org.uk|access-date=2019-06-14}}
History
This drug was developed by Ultragenyx and is in a collaborative license agreement with Kyowa Hakko Kirin.{{Cite press release|title=Collaboration with Ultragenyx to Develop and Commercialize KRN23 for X-linked Hypophosphatemia|date=4 September 2013|publisher=Kyowa Kirin|url=http://www.kyowa-kirin.com/news_releases/2013/e20130904_01.html|access-date=2018-04-17}}
References
{{Reflist}}
External links
- {{cite web | url = https://druginfo.nlm.nih.gov/drugportal/name/burosumab | publisher = U.S. National Library of Medicine | work = Drug Information Portal | title = Burosumab }}
{{monoclonals for bone, musculoskeletal, circulatory, and neurologic systems}}
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