Ultragenyx Pharma Gets EU Orphan Drug Designation For KRN23

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) announced that the European Commission has granted orphan medicinal product designation for recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 or KRN23 or UX023 for the treatment of X-linked hypophosphatemia or XLH.

XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness.

KRN23 is being developed under a license and collaboration agreement between Ultragenyx and Kyowa Hakko Kirin Co., Ltd, Ultragenyx stated.

According to the company, multiple clinical studies in adult patients with XLH have been completed and a Phase 2 study in pediatric patients with XLH is ongoing. The U.S. Food and Drug Administration granted orphan drug designation for KRN23 in XLH in 2009.

The European Commission grants orphan drug status for medicinal products intended to treat diseases or conditions that affect fewer than five in 10,000 people in the European Union.