Abstract
Triheptanoin (DOJOLVI™), a synthetic medium odd-chain (C7) triglyceride, is an effective and generally well tolerated source of calories and fatty acids for the treatment of paediatric and adult patients with molecularly-confirmed long-chain fatty acid oxidation disorders (LC-FAODs). The beneficial effects of triheptanoin are assumed to be linked to the anaplerotic properties of triheptanoin, which set it apart from the standard medium-chain triglyceride (MCT) oil. In a pivotal, randomized, phase 2 trial comparing triheptanoin to trioctanoin in patients with nonsevere LC-FAODs and normal cardiovascular function at baseline, patients in both treatment groups had similar mean changes from baseline in measures of cardiac function and structure after 4 months’ treatment. In patients with severe LC-FAODs, 78-weeks’ treatment with triheptanoin reduced pretreatment mean annualized event and duration rates for major clinical events in an open-label phase 2 trial (CL201). The therapeutic effect of triheptanoin appeared to persist during longer-term therapy in a long-term extension study (CL202). The most frequently reported adverse events were gastrointestinal.
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