Burosumab Explained

Type:mab
Mab Type:mab
Source:u
Target:FGF 23
Pronounce:bur OH sue mab
Tradename:Crysvita
Licence Eu:yes
Dailymedid:Burosumab
Pregnancy Au:B3
Pregnancy Au Comment:[1] [2]
Routes Of Administration:Subcutaneous
Atc Prefix:M05
Atc Suffix:BX05
Legal Au:S4
Legal Au Comment:[3] [4]
Legal Ca:Rx-only
Legal Ca Comment:/ Schedule D[5] [6]
Legal Uk:POM
Legal Uk Comment:[7]
Legal Us:Rx-only
Legal Us Comment:[8] [9]
Legal Eu:Rx-only
Legal Status:Rx-only
Elimination Half-Life:16.4 days[10]
Cas Number:1610833-03-8
Drugbank:DB14012
Chemspiderid:none
Unii:G9WJT6RD29
Kegg:D10913
Synonyms:KRN-23, KRN23, burosumab-twza
C:6388
H:9904
N:1700
O:2006
S:46

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.[11] [12]

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.[13]

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.[14] 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.

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 .[15]

In April 2018, the U.S. Food and Drug Administration (FDA) approved burosumab for its intended purpose in patients aged one year and older.[16] The FDA approval fell under both the breakthrough therapy and orphan drug designations.[17] The FDA considered it to be a first-in-class medication.[18]

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[19] but as of 2019 the drug was available through a simple discount scheme.[20]

History

This drug was developed by Ultragenyx and is in a collaborative license agreement with Kyowa Hakko Kirin.[21]

External links

Notes and References

  1. Web site: Crysvita . Therapeutic Goods Administration (TGA) . 17 September 2021 . 17 September 2021.
  2. Web site: Updates to the Prescribing Medicines in Pregnancy database . Therapeutic Goods Administration (TGA) . 12 May 2022 . 13 May 2022.
  3. Web site: Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial . Therapeutic Goods Administration (TGA) . 17 September 2021 . 17 September 2021 . https://web.archive.org/web/20210917193625/https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 . dead .
  4. Web site: Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial . Therapeutic Goods Administration (TGA) . PDF . 17 September 2021 . 17 September 2021 . https://web.archive.org/web/20210917193625/https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793 . dead .
  5. Web site: Crysvita Product information . Health Canada . 25 April 2012 . 29 May 2022.
  6. Web site: Summary Basis of Decision (SBD) for Crysvita . . 23 October 2014 . 29 May 2022.
  7. Web site: Crysvita 10 mg solution for injection - Summary of Product Characteristics (SmPC) . (emc) . 20 April 2020 . 19 June 2020.
  8. Web site: Crysvita- burosumab injection . DailyMed . 17 September 2021.
  9. Web site: Drug Approval Package: Crysvita (burosumab-twza) . U.S. Food and Drug Administration (FDA) . 15 May 2018 . 28 February 2020.
  10. Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey J, Glorieux FH, Portale AA, Insogna K, Carpenter TO, Peacock M . 6 . Pharmacokinetics and pharmacodynamics of a human monoclonal anti-FGF23 antibody (KRN23) in the first multiple ascending-dose trial treating adults with X-linked hypophosphatemia . Journal of Clinical Pharmacology . 56 . 2 . 176–85 . February 2016 . 26073451 . 5042055 . 10.1002/jcph.570 .
  11. ((World Health Organization)) . 2017 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77 . WHO Drug Information . 31 . 1 . 10665/330984 . free .
  12. Web site: Burosumab (KRN23) for X-Linked Hypophosphatemia (XLH). . n.d.. 2018-04-18. https://web.archive.org/web/20180418230050/http://www.c-hotline.net/docs/html/KYOW7605/dl/kyow171027_1.pdf. 2018-04-18. dead.
  13. Web site: What Is X-Linked Hypophosphatemia? . Ultragenyx Pharmaceutical . 22 February 2020 . 23 June 2021 . https://web.archive.org/web/20210623201520/https://www.ultragenyx.com/patients/xlh/ . dead .
  14. FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening . U.S. Food and Drug Administration . 18 June 2020 . 19 June 2020.
  15. Web site: Crysvita EPAR . European Medicines Agency (EMA) . 17 September 2018 . 1 March 2020.
  16. FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia. 17 April 2018. U.S. Food and Drug Administration (FDA).
  17. Web site: Crysvita Orphan Drug Designation . U.S. Food and Drug Administration (FDA) . 24 December 1999 . 27 February 2020.
  18. New Drug Therapy Approvals 2018 . U.S. Food and Drug Administration (FDA) . January 2019 . PDF . 16 September 2020.
  19. News: U.K. cost watchdogs turn away rare disease med Crysvita. Fierce Pharma . 15 June 2018.
  20. Web site: 1 Recommendations Burosumab for treating X-linked hypophosphataemia in children and young people. Guidance NICE. www.nice.org.uk. 2019-06-14.
  21. Collaboration with Ultragenyx to Develop and Commercialize KRN23 for X-linked Hypophosphatemia. 4 September 2013. Kyowa Kirin. 2018-04-17.

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