KRN 23 (monoclonal antibody) filed with the EU for the treatment of X-linked hypophosphatemia- Ultragenyx Pharmaceutical / Kyowa Kirin

Ultragenyx Pharmaceutical and Kyowa Kirin International announced that the European Medicines Agency has accepted for review the Marketing Authorisation Application (MAA) for KRN 23 (monoclonal antibody) for the treatment of X-linked hypophosphatemia. The MAA was filed and accepted in late 2016, and an opinion from the Committee for Medicinal Products for Human Use is expected in the second half of 2017.

The application is based on the positive results from multiple studies in paediatric and adult patients with XLH. A Phase III programme studying KRN 23 in adults and Phase II and Phase III studies in paediatric patients with XLH are ongoing. KRN 23 is also being developed for tumour-induced osteomalacia (TIO), a disease characterised by typically benign tumours that produce excess levels of FGF23, which can lead to severe osteomalacia, fractures, bone and muscle pain, and muscle weakness. Ultragenyx and Kyowa Hakko Kirin plan to submit a biologics licence application to the U.S. FDA for KRN 23 in the second half of 2017.