Ultragenyx and Kyowa Kirin International Announce Marketing Authorisation Application

Dylan Macdonaldblog

Ultragenyx and Kyowa Kirin International Announce Marketing Authorisation Application for KRN23 Filed and Accepted for Review by European Medicines Agency. Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Kyowa Kirin International PLC (KKI), a wholly owned subsidiary of Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin), today announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorisation Application (MAA) for KRN23 for the treatment of X-linked hypophosphatemia (XLH). The MAA was filed and accepted in late 2016, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the second half of 2017. Ultragenyx and Kyowa Hakko Kirin entered into a collaboration and licence agreement in August 2013 to develop and commercialise KRN23.

“Based on the positive results from multiple studies in paediatric and adult patients with XLH, we are pursuing conditional marketing authorisation in order to accelerate access to this therapy for patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.

“X-linked hypophosphatemia is a debilitating condition which causes long term pain and distress among sufferers and for which there are no currently approved treatments that target the underlying cause,” said Dr. Tom Stratford, President and CEO of KKI. “As part of Kyowa Hakko Kirin, we strive to contribute to the health and wellbeing of people around the world through advances in life sciences and technologies. The acceptance of this filing brings us one step closer to addressing the unmet medical needs of patients who suffer from X-linked hypophosphatemia.”