triheptanoin

Triheptanoin is a synthetic, odd-carbon, medium-chain triglyceride (C7) that serves as a substrate replacement therapy and anaplerotic agent for rare metabolic and neurological disorders. Developed by Ultragenyx Pharmaceutical and licensed from Baylor Research Institute, it is metabolized into heptanoate and subsequently into C5 ketone bodies (beta-ketopentanoate and beta-hydroxypentanoate) and propionyl-CoA. Unlike common even-chain fats, triheptanoin's metabolism provides intermediates that directly replenish the tricarboxylic acid (TCA) cycle, supporting energy production in patients with metabolic blocks. It was approved by the FDA in June 2020 under the brand name Dojolvi for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). It has also been clinically evaluated for glucose transporter type-1 deficiency syndrome (Glut1 DS) and drug-resistant epilepsy, where it aims to provide an alternative energy source to the glucose-deprived brain.

Brand names

Dojolvi

Other names

triheptanointriglyceride of heptanoateglycerol triheptanoateUX007UX-007UX 007