Ultragenyx Pharma Receives Breakthrough Therapy Designation For KRN23

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) announced it has received Breakthrough Therapy Designation from the U.S. FDA for KRN23 for the treatment of X-linked hypophosphatemia in pediatric patients one year of age and older. The Breakthrough Therapy Designation is based on interim 40-week data from the first 36 patients enrolled in the ongoing pediatric Phase 2 study of KRN23.

In addition to the ongoing Phase 2 study, Ultragenyx plans to initiate a Phase 3 study of KRN23 in pediatric patients in mid-2016. A Phase 3 program of KRN23 in adult patients with X-linked hypophosphatemia is ongoing.