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]
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]
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]
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.[16]
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 .[17]
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][18] The FDA considered it to be a first-in-class medication.[19]
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[20] but as of 2019 the drug was available through a simple discount scheme.[21]
This drug was developed by Ultragenyx and is in a collaborative license agreement with Kyowa Hakko Kirin.[22]
Lokasi Pengunjung: 18.117.172.186