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Burosumab

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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
Pharmacokinetic data
Elimination half-life16.4 days[10]
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][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.[13] 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.[14] Thus, burosumab is designed to bind to the FGF23 receptor and inhibit the excess activity of the FGF23 hormone within the body.[medical citation needed]

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.[15] The tumors associated with TIO release fibroblast growth factor 23 (FGF23) which lowers phosphate levels.[15]

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.[13] The FDA approval fell under both the breakthrough therapy and orphan drug designations.[13][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. ^ a b "Crysvita". Therapeutic Goods Administration (TGA). 17 September 2021. Retrieved 17 September 2021.
  2. ^ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 12 May 2022. Retrieved 13 May 2022.
  3. ^ "Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial". Therapeutic Goods Administration (TGA). Archived from the original on 17 September 2021. Retrieved 17 September 2021.
  4. ^ "Crysvita burosumab 10 mg/mL solution for injection in a 5 mL vial". Therapeutic Goods Administration (TGA). Archived from the original (PDF) on 17 September 2021. Retrieved 17 September 2021.
  5. ^ "Crysvita Product information". Health Canada. 25 April 2012. Retrieved 29 May 2022.
  6. ^ "Summary Basis of Decision (SBD) for Crysvita". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  7. ^ "Crysvita 10 mg solution for injection - Summary of Product Characteristics (SmPC)". (emc). 20 April 2020. Retrieved 19 June 2020.
  8. ^ a b "Crysvita- burosumab injection". DailyMed. Retrieved 17 September 2021.
  9. ^ "Drug Approval Package: Crysvita (burosumab-twza)". U.S. Food and Drug Administration (FDA). 15 May 2018. Retrieved 28 February 2020.
  10. ^ Zhang X, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Ito T, et al. (February 2016). "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. doi:10.1002/jcph.570. PMC 5042055. PMID 26073451.
  11. ^ World Health Organization (2017). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77". WHO Drug Information. 31 (1). hdl:10665/330984.
  12. ^ "Burosumab (KRN23) for X-Linked Hypophosphatemia (XLH)" (PDF). n.d. Archived from the original (PDF) on 18 April 2018. Retrieved 18 April 2018.
  13. ^ a b c "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. Public Domain This article incorporates text from this source, which is in the public domain.
  14. ^ "What Is X-Linked Hypophosphatemia?". Ultragenyx Pharmaceutical. Archived from the original on 23 June 2021. Retrieved 22 February 2020.
  15. ^ a b "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. Public Domain This article incorporates text from this source, which is in the public domain.
  16. ^ "Crysvita EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 1 March 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  17. ^ "Crysvita Orphan Drug Designation". U.S. Food and Drug Administration (FDA). 24 December 1999. Retrieved 27 February 2020.
  18. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
  19. ^ "U.K. cost watchdogs turn away rare disease med Crysvita". Fierce Pharma. 15 June 2018.
  20. ^ "1 Recommendations | Burosumab for treating X-linked hypophosphataemia in children and young people. Guidance NICE". www.nice.org.uk. Retrieved 14 June 2019.
  21. ^ "Collaboration with Ultragenyx to Develop and Commercialize KRN23 for X-linked Hypophosphatemia" (Press release). Kyowa Kirin. 4 September 2013. Retrieved 17 April 2018.
  • "Burosumab". Drug Information Portal. U.S. National Library of Medicine.
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