Chemistry:Burosumab

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Short description: Monoclonal antibody designed to treat X-linked hypophosphatemia
Burosumab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetFGF 23
Clinical data
Pronunciationbur OH sue mab
Trade namesCrysvita
Other namesKRN-23, KRN23, burosumab-twza
AHFS/Drugs.comMonograph
MedlinePlusa618034
License data
Pregnancy
category
Routes of
administration		Subcutaneous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6388H9904N1700O2006S46
Molar mass144090.15 g·mol−1

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.[8][10][11]

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.[12] 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] Thus, burosumab is designed to bind to the FGF23 receptor and inhibit the excess activity of the FGF23 hormone within the body.

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.[14]

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.[15]

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 .[16]

In April 2018, the U.S. Food and Drug Administration (FDA) approved burosumab for its intended purpose in patients aged one year and older.[12] The FDA approval fell under both the breakthrough therapy and orphan drug designations.[12][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]

References

  1. ↑ 1.0 1.1 "Crysvita". 17 September 2021. https://www.tga.gov.au/apm-summary/crysvita. 
  2. ↑ "Updates to the Prescribing Medicines in Pregnancy database". 12 May 2022. https://www.tga.gov.au/resources/resource/guidance/updates-prescribing-medicines-pregnancy-database. 
  3. ↑ "Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial". https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793. 
  4. ↑ "Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial" (PDF). https://tga-search.clients.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=340793. 
  5. ↑ "Crysvita Product information". 25 April 2012. https://health-products.canada.ca/dpd-bdpp/info.do?lang=en&code=97399. 
  6. ↑ "Summary Basis of Decision (SBD) for Crysvita". 23 October 2014. https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00429&lang=en. 
  7. ↑ "Crysvita 10 mg solution for injection - Summary of Product Characteristics (SmPC)". 20 April 2020. https://www.medicines.org.uk/emc/product/9779/smpc. 
  8. ↑ 8.0 8.1 "Crysvita- burosumab injection". https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6f5f3556-3197-477a-993c-96b2c29a91ac. 
  9. ↑ "Drug Approval Package: Crysvita (burosumab-twza)". 15 May 2018. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/761068Orig1s000TOC.cfm. 
  10. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77". WHO Drug Information 31 (1). 2017. 
  11. ↑ "Burosumab (KRN23) for X-Linked Hypophosphatemia (XLH)". n.d.. http://www.c-hotline.net/docs/html/KYOW7605/dl/kyow171027_1.pdf. 
  12. ↑ 12.0 12.1 12.2 "FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia" (Press release). U.S. Food and Drug Administration (FDA). 17 April 2018. This article incorporates text from this source, which is in the public domain.
  13. ↑ "What Is X-Linked Hypophosphatemia?". Ultragenyx Pharmaceutical. http://www.ultragenyx.com/patients/xlh/. 
  14. ↑ 14.0 14.1 "FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening". U.S. Food and Drug Administration (Press release). 18 June 2020. Retrieved 19 June 2020. This article incorporates text from this source, which is in the public domain.
  15. ↑ FDA Professional Drug Information
  16. ↑ "Crysvita EPAR". 17 September 2018. https://www.ema.europa.eu/en/medicines/human/EPAR/crysvita.  This article incorporates text from this source, which is in the public domain.
  17. ↑ "Crysvita Orphan Drug Designation". 24 December 1999. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=296109. 
  18. ↑ (PDF) New Drug Therapy Approvals 2018 (Report). January 2019. https://www.fda.gov/media/120357/download. Retrieved 16 September 2020. 
  19. ↑ "U.K. cost watchdogs turn away rare disease med Crysvita". Fierce Pharma. 15 June 2018. https://www.fiercepharma.com/pharma/u-k-cost-watchdogs-turn-away-rare-disease-med-crysvita. 
  20. ↑ "1 Recommendations | Burosumab for treating X-linked hypophosphataemia in children and young people. Guidance NICE". https://www.nice.org.uk/guidance/hst8/chapter/1-Recommendations. 
  21. ↑ "Collaboration with Ultragenyx to Develop and Commercialize KRN23 for X-linked Hypophosphatemia" (Press release). Kyowa Kirin. 4 September 2013. Retrieved 2018-04-17.

External links



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