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

News | 10. 27. 2014

Ultragenyx Pharmaceutical

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.