Ultragenyx's burosumab gets Priority Review for XLH

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) said FDA accepted and granted Priority Review to a BLA for burosumab (KRN23, UX023) to treat X-linked hypophosphatemia (XLH) in adult and pediatric patients. The PDUFA date is April 17, 2018.

The BLA is based in part on a 24-week Phase III trial of burosumab in adults with XLH. Burosumab met the study's primary endpoint of improving serum phosphorus response, with 94% of patients achieving and maintaining normal levels of serum phosphorus compared to 8% on placebo (p<0.0001) (see BioCentury Extra, April 18)...