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Triheptanoin in the management of long-chain fatty acid oxidation disorders: a profile of its use

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posted on 21.03.2021, 19:24 by Esther S. Kim, Susan J. Keam

Declarations


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.


Additional information about this Adis Drug Review can be found here


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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