Burosumab recommended for Rare muscoskeletal disorder

Kyowa Hakko Kirin Co., Ltd., Kyowa Kirin International PLC and Ultragenyx Pharmaceutical Inc. announced that the Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency's (EMA) scientific committee, has adopted a Positive Opinion recommending the conditional marketing authorization of burosumab, an anti-FGF23 human monoclonal antibody, for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

XLH is a rare, genetic, chronic and progressive musculoskeletal disorder. If approved, burosumab would be the first therapy that addresses the excess FGF23 activity in XLH. The CHMP's opinion is now being referred to the European Commission (EC), for a final decision on the grant of a conditional marketing authorization. This decision is expected in the first quarter of 2018 and will apply to all 28 countries of the European Union, Norway, Iceland and Liechtenstein. The conditional authorization requires fulfilment of specific obligations related to the completion of ongoing clinical studies of burosumab in pediatric patients.