Burosumab Biologics License Application Accepted by FDA and Granted Priority Review

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin) and Kyowa Kirin International PLC (Kyowa Kirin International) have announce that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for burosumab to treat pediatric and adult patients with X-Linked Hypophosphatemia (XLH) and has granted Priority Review status.

Burosumab previously received Breakthrough Therapy Designation from the FDA for the treatment of XLH in pediatric patients one year of age and older.

The FDA has granted burosumab Priority Review status, which is available to drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. The Prescription Drug User Fee Act (PDUFA) action date for the BLA is April 17, 2018. The Agency has not informed the companies whether an advisory committee meeting to discuss the application will be needed. The FDA previously designated burosumab as a drug for a “rare pediatric disease”, enabling issuance of a priority review voucher if burosumab is approved.

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"XLH is a debilitating disease and there are no current treatment options that address the underlying cause,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We are pleased that the FDA has granted priority review and are looking forward to working with the agency in the coming months with the goal of bringing this potential new treatment to patients as quickly as possible.”

“Burosumab is the culmination of Kyowa Hakko Kirin’s research activity in diseases in which high levels of FGF 23 are a causative factor,” said Mitsuo Satoh, Executive Officer, Vice President, Head of Research and Development Division of Kyowa Hakko Kirin. “I believe burosumab has the potential to be an effective treatment option for patients with conditions with excess FGF23 such as XLH and tumor induced osteomalacia and we will keep working to provide this advance in therapy for patients as soon as possible.”

Dr. Tom Stratford, President and Chief Executive of Kyowa Kirin International, said “Burosumab has the potential to treat both pediatric and adult patients with XLH and we welcome the fact that the FDA has granted burosumab priority review status, meaning that sufferers may be able to have access to this treatment earlier.”

Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin, have been collaborating in the development and commercialization of burosumab globally, based on the collaboration and license agreement between Kyowa Hakko Kirin and Ultragenyx.