FDA Not Currently Planning to Hold Advisory Committee Meeting for Burosumab Biologics License Application

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co., Ltd. and Kyowa Kirin International PLC have announced that the U.S. Food and Drug Administration (FDA) has indicated in the “Day 74” filing communication that it is not currently planning to hold an advisory committee meeting to discuss the Biologics License Application (BLA) for burosumab to treat pediatric and adult patients with X-Linked Hypophosphatemia (XLH).

Additionally, the FDA indicated that the review is proceeding according to its internal review timelines described in their Guidance on Good Review Management Principles and Practices for PDUFA Products.

“We are working closely with the FDA as they review the burosumab application, and are pleased with our ongoing interactions with the FDA and its progress in reviewing the BLA,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.

“We are pleased with this progress in reviewing the BLA for burosumab, and will keep working to provide this new therapy for patients as soon as possible.” said, Masashi Miyamoto, Ph.D., Director of the Board, Managing Executive Officer, Director of Corporate Strategy & Planning Department of Kyowa Hakko Kirin.

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The FDA previously 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 FDA has designated burosumab as a drug for a “rare pediatric disease”, enabling issuance of a priority review voucher if burosumab is approved. Burosumab previously received Breakthrough Therapy Designation from the FDA for the treatment of XLH in pediatric patients one year of age and older.

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.

About Burosumab

Burosumab is an investigational recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko Kirin, against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Burosumab is being developed to treat XLH and tumor-induced osteomalacia (TIO), diseases characterized by excess levels of FGF23. Phosphate wasting in XLH and TIO is caused by excessive levels and activity of FGF23. Burosumab is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with XLH and TIO, burosumab is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

A clinical program studying burosumab in adults and pediatric patients with XLH is ongoing. Burosumab 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.

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