ARTICLE | Clinical News

Triheptanoin regulatory update

November 10, 2014 8:00 AM UTC

FDA granted Orphan Drug designation to triheptanoin from Ultragenyx to treat glucose transporter type-1 deficiency syndrome (De Vivo disease). The product is in Phase II testing for the indication in patients who failed a ketogenic diet and who continue to have breakthrough seizures. De Vivo disease is associated with a deficiency of solute carrier family 2 facilitated glucose transporter member 1 ( SLC2A1; GLUT1) and characterized by seizures, developmental delay and movement disorder. Triheptanoin is a synthetic compound that provides medium-length, odd-chain fatty acids that are metabolized to replace intermediate substrates in fatty acid oxidation and in the tricarboxylic acid (TCA) cycle. ...