Kyowa Kirin announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the expanded approval of Crysvita (burosumab) to include older adolescents and adults living with the rare disease X-linked hypophosphataemia (XLH). The European Commission has already granted a conditional marketing authorisation for Crysvita for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons.
The CHMP recommends that this approval is expanded to include all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults with XLH. The CHMP opinion will now be reviewed by the European Commission, with a final decision expected in September 2020.
The positive opinion from the CHMP was based on data from two Phase III studies: the Phase III UX023-CL303 study, a randomised, double-blind, placebo-controlled trial investigating the safety and efficacy of burosumab in adults with XLH, and the Phase III UX023-CL304 study, an open-label, single-arm trial investigating the effects of burosumab on osteomalacia in adults with XLH.