Burosumab

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Burosumab
Monoclonal antibody
TypeWhole antibody
SourceHuman
TargetFGF 23
Names
Pronunciationbur OH sue mab
Trade namesCrysvita
Other namesKRN-23, KRN23, burosumab-twza
Clinical data
Main usesX-linked hypophosphatemia, tumor-induced osteomalacia[1]
Side effectsFever, pain at the site of injection, cough, headache, restless leg syndrome, cavities, tooth abscess[1]
Pregnancy
category
Routes of
use
Subcutaneous
External links
AHFS/Drugs.comMonograph
US NLMBurosumab
MedlinePlusa618034
Legal
License data
Legal status
Pharmacokinetics
Elimination half-life16.4 days[7]
Chemical and physical data
FormulaC6388H9904N1700O2006S46
Molar mass144090.15 g·mol−1

Burosumab, sold under the brand name Crysvita, is a medication used to treat X-linked hypophosphatemia and tumor-induced osteomalacia.[1] It may be used in those over 5 months old.[1] It is given by injection under the skin.[8]

Common side effects include fever, pain at the site of injection, cough, headache, restless leg syndrome, cavities, and tooth abscess.[1] Other side effects may include allergic reactions, high phosphate, and nephrocalcinosis.[1] Safety in pregnancy is unclear.[1] It is a monoclonal antibody that binds to and blocks the protein FGF23, allowing the kidneys to reabsorb phosphate.[9]

Burosumab was approved for medical use in the United States and Europe in 2018.[1][9] In the United States 30 mg costs about 12,200 USD as of 2022.[10]

Medical uses

In 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,[11] and for the treatment of people age two and older with tumor-induced osteomalacia (TIO), a rare disease that is characterized by the development of tumors that cause weakened and softened bones.[12] The tumors associated with TIO release a peptide hormone-like substance known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.[12]

In the European Union, burosumab is indicated for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons.[9]

XLH is genetic disorder affecting phosphate metabolism within the body, which results in hypophosphatemia. The disease is characterized by overproduction of the FGF23 hormone in bone cells. The FGF23 hormone is responsible for blocking phosphate re-absorption by the kidney and the suppression of the vitamin D dependent phosphate absorption by the intestine. Due to the excess activity of FGF23, phosphate levels in the blood are abnormally low, 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.[medical citation needed]

While burosumab is effective for the treatment of X-linked hypophosphatemia, the National Institute for Health and Care Excellence in England and Wales initially raised concerns regarding the incremental cost-effectiveness of the new treatment[14] but the drug is available through a simple discount scheme.[15]

Dosage

The dose in adults is 1 mg/kg of body weight, up to a maximum dose of 90 mg every four weeks.[9] The dose in children is 0.8 mg/kg of body weight, up to a maximum dose of 90 mg every two weeks.[9]

History

This drug was developed by Ultragenyx and is in a collaborative license agreement with Kyowa Hakko Kirin.[16]

The FDA approval fell under both the breakthrough therapy and orphan drug designations.[11][17] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[18]

References

  1. 1.0 1.1 1.2 1.3 1.4 1.5 1.6 1.7 1.8 "Crysvita- burosumab injection". DailyMed. Archived from the original on 17 September 2021. Retrieved 17 September 2021.
  2. 2.0 2.1 "Crysvita". Therapeutic Goods Administration (TGA). 17 September 2021. Archived from the original on 17 September 2021. Retrieved 17 September 2021.
  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" (PDF). Therapeutic Goods Administration (TGA). Archived from the original on 17 September 2021. Retrieved 17 September 2021.
  5. "Crysvita 10 mg solution for injection - Summary of Product Characteristics (SmPC)". (emc). 20 April 2020. Archived from the original on 21 June 2020. Retrieved 19 June 2020.
  6. "Drug Approval Package: Crysvita (burosumab-twza)". U.S. Food and Drug Administration (FDA). 15 May 2018. Archived from the original on 28 February 2020. Retrieved 28 February 2020.
  7. 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.
  8. "Burosumab". SPS - Specialist Pharmacy Service. 30 November 2016. Archived from the original on 22 June 2020. Retrieved 12 January 2022.
  9. 9.0 9.1 9.2 9.3 9.4 "Crysvita EPAR". European Medicines Agency (EMA). 17 September 2018. Archived from the original on 25 July 2021. Retrieved 1 March 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  10. "Crysvita Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 14 April 2021. Retrieved 12 January 2022.
  11. 11.0 11.1 "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. Archived from the original on 9 October 2021. Retrieved 18 September 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  12. 12.0 12.1 "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. Archived from the original on 26 July 2021. Retrieved 19 June 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  13. "What Is X-Linked Hypophosphatemia?". Ultragenyx Pharmaceutical. Archived from the original on 23 June 2021. Retrieved 18 September 2021.
  14. "U.K. cost watchdogs turn away rare disease med Crysvita". Archived from the original on 24 January 2021. Retrieved 18 September 2021.
  15. "1 Recommendations | Burosumab for treating X-linked hypophosphataemia in children and young people | Guidance | NICE". www.nice.org.uk. Archived from the original on 4 November 2021. Retrieved 14 June 2019.
  16. "Collaboration with Ultragenyx to Develop and Commercialize KRN23 for X-linked Hypophosphatemia" (Press release). Kyowa Kirin. 4 September 2013. Archived from the original on 18 December 2018. Retrieved 17 April 2018.
  17. "Crysvita Orphan Drug Designation". U.S. Food and Drug Administration (FDA). 24 December 1999. Archived from the original on 4 November 2021. Retrieved 27 February 2020.
  18. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.

External links

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