Triheptanoin for long chain fatty acid oxidation disorders

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Year: 2020

Triheptanoin (UX007) is being developed for the treatment of long chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is a group of six rare 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 hospitalizations 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.
Triheptanoin is an orally administered synthetic (artificially produced) fat, which is broken down in the liver into substances that can be used to generate energy. Triheptanoin could provide a source of calories and fatty acids for patients with LC-FAOD, potentially improving their muscle function, exercise tolerance, and health-related quality of life. If licenced, triheptanoin could provide a treatment option for paediatric and adult patients with molecularly confirmed LC-FAOD who currently have no approved therapies.