![From QSR to QMSR: Meeting FDA’s New Requirements From QSR to QMSR: Meeting FDA’s New Requirements](https://www.fdanews.com/ext/resources/Book-Covers-NEW/From-QSR-to-QMSR-500.png?height=200&t=1714569011&width=200)
Home » FDA Approves First Treatment for Rare Form of Rickets
FDA Approves First Treatment for Rare Form of Rickets
![FDA_Logo_Blue_2016.gif](https://www.fdanews.com/ext/resources/test/Drug-Images4/FDA_Logo_Blue_2016.gif?t=1578316591&width=430)
April 18, 2018
The FDA approved Crysvita (burosumab twza), marking the first therapy cleared by the agency for treating X-linked hypophosphatemia (XLH) in adults and children 1 years of age or older.
The antibody therapy treats XLH, a rare and inherited form of rickets, by blocking fibroblast growth factor 23, a hormone that triggers phosphate urinary excretion and stifles the kidney’s active vitamin D production.
The safety and efficacy of the drug was determined through four clinical trials. In the placebo trials, 94 percent of adults receiving the treatment once a month achieved normal phosphorus levels compared to 8 percent receiving the placebo.
Upcoming Events
-
21Oct