Ultragenyx Granted EU Orphan Drug Designation for KRN23 for the Treatment of X-Linked Hypophosphatemia

News | 10. 30. 2014

Ultragenyx Pharmaceutical

Novato, CA, Oct. 30, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the European Commission has granted orphan medicinal product designation for recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (KRN23 or UX023) for the treatment of X-linked hypophosphatemia (XLH). XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness.