Posted by Michael Wonder on 17 Apr 2018
FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphataemia
17 April 2018 - The U.S. FDA today approved Crysvita (burosumab), the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets.
XLH causes low levels of phosphorus in the blood. It leads to impaired bone growth and development in children and adolescents and problems with bone mineralization throughout a patient’s life.
The safety and efficacy of Crysvita were studied in four clinical trials. In the placebo-controlled trial, 94% of adults receiving Crysvita once a month achieved normal phosphorus levels compared to 8% of those receiving placebo. In children, 94 to 100% of patients treated with Crysvita every two weeks achieved normal phosphorus levels. In both children and adults, X-ray findings associated with XLH improved with Crysvita therapy. Comparison of the results to a natural history cohort also provided support for the effectiveness of Crysvita.
