Triheptanoin

(Dojolvi®)

Triheptanoin

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Drug updated on 5/17/2024

Dosage FormLiquid (oral; 100% w/w of triheptanoin)
Drug ClassMedium-chain triglycerides
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

Summary
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  • Triheptanoin (Dojolvi) is indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
  • Five randomized controlled studies focused on its efficacy in treating LC-FAODs.
  • In these studies, triheptanoin demonstrated significant efficacy by reducing major clinical events such as rhabdomyolysis, hypoglycemia, and cardiomyopathy across different patient cohorts.
  • The safety profile of triheptanoin was consistent with previous observations; most adverse events were mild to moderate in severity.
  • It did not show significant improvement in heart rate during exercise or exercise performance for patients with phosphofructokinase deficiency nor significantly reduce seizure frequency for those with glucose transporter 1 deficiency syndrome.
  • The studies highlight that Dojolvi's effectiveness might vary among different patient subtypes within a disorder spectrum.

Product Monograph / Prescribing Information

Document TitleYearSource
Dojolvi (triheptanoin) Prescribing Information.2023Ultragenyx Pharmaceutical Inc., Novato, CA

Randomized Controlled Trials

Clinical Practice Guidelines