Triheptanoin for long-chain fatty acid oxidation disorders

Triheptanoin is being developed for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is a group of six rare and ultra-rare life-threatening genetic disorders in which the body is unable to convert dietary fatty acids into energy. This inability to produce energy from fat can lead to severe depletion of glucose in the body and serious, unpredictable complications, which can lead to hospitalisations or early death despite the best current care. LC-FAOD has no specifically approved treatments. The current disease management includes avoidance of fasting, maintenance of a low-fat diet, and supplementation of diet with oils rich in essential fatty acids.