EU approval for Crysvita to treatX-linked hypophosphatemia .- Kyowa Hakko Kirin + Ultragenyx.

Kyowa Hakko Kirin Co. Ltd, and Ultragenyx Pharmaceutical Inc. announced that Crysvita (burosumab) has received a positive European Commission decision granting a conditional marketing authorization to Kyowa Kirin 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, chronic progressive musculoskeletal disorder that affects children and adults.

This is the first regulatory approval globally for Crysvita, an anti-FGF23 fully human monoclonal antibody that is the first treatment to target the underlying pathophysiology of XLH. The European Medicines Agency recently acknowledged Crysvita as an outstanding contribution to public health and a significant improvement in the endocrinology therapeutic area. The European Marketing Authorization is valid in the 28 countries of the European Union and in Norway, Iceland and Liechtenstein. The first commercial launch of Crysvita is expected to take place in Germany in the second quarter of 2018, followed by other European countries.

In the United States, the FDA is currently reviewing the Biologics License Application for Crysvita to treat pediatric and adult patients with XLH, and has set a Prescription Drug User Fee Act (PDUFA) action date of 17 April 2018.

A clinical program studying Crysvita in adults and pediatric patients with XLH is ongoing. Crysvita is also being developed for TIO, a disease characterized by typically benign tumors that produce excess levels of FGF23, which can lead to severe osteomalacia, fractures, bone and muscle pain, and muscle weakness.