FDA Approves First Treatment for Long-Chain Fatty Acid Oxidation Disorders

The Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin; Ultragenyx) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). 

Triheptanoin is a medium-chain triglyceride that bypasses the genetic block in long-chain fatty acid metabolism and provides a source of calories and fatty acids to patients with LC-FAOD. The approval was based on data from multiple studies, including a 4-month, randomized, double-blind phase 2 study that compared the efficacy of triheptanoin to trioctanoin (8-carbon chain fatty acid) in 32 patients aged ≥7 years with a confirmed diagnosis of LC-FAOD. The primary outcomes measures of the trial included energy expenditure and change in resting ejection fraction.

Results showed that after 4 months, both treatment arms had similar mean changes from baseline in left ventricular ejection fraction and wall mass on resting echocardiogram, as well as similar maximal heart rates on treadmill ergometry (secondary end point). As for safety, the most common adverse reactions reported with triheptanoin were abdominal pain, diarrhea, vomiting, and nausea.

Additionally, an open-label phase 2 study assessing the safety and clinical effects of triheptanoin in 29 patients showed that treatment led to a 48% reduction in the mean annualized rate of major clinical events (aggregate of events related to hypoglycemia, cardiomyopathy, and rhabdomyolysis).

Dojolvi is expected to be available in the next 30 days. The product will be supplied as an oral liquid in 500mL bottles containing 100% w/w of triheptanoin. The caloric value of triheptanoin is 8.3kcal/mL. 

For more information visit ultragenyx.com.