Ultragenyx Pharma Gets Orphan Drug Designation For Triheptanoin - Quick Facts

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) reported that the FDA Office of Orphan Products Development has granted orphan drug designation for triheptanoin (UX007) to treat glucose transporter type-1 deficiency syndrome or Glut1 DS.

Glut1 DS, also referred to 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.

According to Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx, "Glut1 deficiency syndrome is a rare condition that can severely affect patients and their families. Ultragenyx is committed to addressing this high unmet medical need population and has initiated a rigorous development plan to do so. The FDA's decision to grant triheptanoin orphan drug designation for Glut1 deficiency syndrome is another important step in our plan to help treat patients with this disease."

The Orphan Drug Designation program offers orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.