Crysvita Approved to Treat Rare Form of Rickets
April 17, 2018 – The U.S. FDA has approved Crysvita® (burosumab), manufactured by Ultragenyx and Kyowa Kirin, to treat adults and children at least one year of age who have a rare, inherited form of rickets known as x-linked hypophosphatemia (XLH).
XLH affects an estimated 3,000 children and 12,000 adults in the United States. Usually diagnosed during childhood, the condition results from a mutation found on the X-chromosome that leads to low phosphate levels in the bloodstream and phosphate wasting. XLH is characterized by weak, soft bones, bowed or bent legs, short stature, dental pain, and bone pain in children. In adults, it can lead to persistent discomfort or complications, including joint pain, poor mobility, tooth abscesses, and hearing loss.
Crysvita blocks fibroblast growth factor 23 (FGF23), which leads to an increase in vitamin D production and in phosphate reabsorption from the kidney. In clinical trials, 94% of adults treated with Crysvita once monthly achieved normal phosphate levels, compared to just 8% of adults who received the placebo. From 94% to 100% of children treated with Crysvita were able to reach normal phosphate levels.
The expected cost for Crysvita after rebates and discounts will range from $160,000 per year for children, and $200,000 per year for adults. Crysvita launched in the United States on April 27, 2018, and will be available in single-dose vials of 10mg/mL, 20mg/mL, and 30mg/mL solution.
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