Triheptanoin in the management of long-chain fatty acid oxidation disorders: a profile of its use
Funding The preparation of this review was not supported by any external funding.
Authorship and conflicts of interest ES Kim, a contracted employee of Adis International Ltd/Springer Nature and SJ Keam, a salaried employee of Adis International Ltd/Springer Nature, declare no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.
Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable.
information about this Adis Drug Review can be found here
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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