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

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

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.

Ultragenyx Granted Orphan Drug Designation for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome

NOVATO, CA – October 27, 2014 – 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 FDA Office of Orphan Products Development has granted orphan drug designation for triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS, also known as De Vivo disease, is a rare and potentially severely debilitating disease characterized by seizures, developmental delay, and movement disorder. Ultragenyx is conducting a Phase 2 study of triheptanoin in patients with Glut1 DS.