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Dojolvi (triheptanoin) is a medium-chain triglyceride.
Dojolvo is specifically indicated 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).
Dojolvi is supplied as an oral liquid. Assess the metabolic requirements of the patient by determining their daily caloric intake (DCI) prior to calculating the dose of Dojolvi. For patients receiving another medium-chain triglyceride (MCT) product, discontinue prior to the first dose of Dojolvi. The recommended target daily dosage of Dojolvi is up to 35% of the patient’s total prescribed DCI divided into at least four doses and administered at mealtimes or with snacks. In order to reach a target daily dosage, patients may require an increase in their total fat intake. All patients treated with Dojolvi should be under the care of a clinical specialist knowledgeable in appropriate disease-related dietary management based upon current nutritional recommendations. The neonatal population may require higher fat intake and therefore an increased amount of Dojolvi. Current nutritional recommendations should be considered when dosing the neonatal population. Please see the drug label to calculate the total daily dosage converted to a volume of DojolviI to be administered in mL. Please see drug label for additional specific recommendations based on prior treatment and drug tolerability.
The efficacy of triheptanoin as a source of calories and fatty acids was evaluated in Study 3, a 4- month double-blind randomized controlled study comparing triheptanoin (7-carbon chain fatty acid) with trioctanoin (8-carbon chain fatty acid). The study enrolled 32 adult and pediatric patients with a confirmed diagnosis of LC-FAOD and evidence of at least one significant episode of rhabdomyolysis and at least two of the following diagnostic criteria: disease specific elevation of acylcarnitines on a new born blood spot or in plasma, low enzyme activity in cultured fibroblasts, or one or more known pathogenic mutations in CPT2, ACADVL, HADHA, or HADHB. The dosage of study drug was titrated to a protocol-specified target of 20% DCI (actual mean daily dose achieved was 16% for triheptanoin and 14% for trioctanoin). The recommended target dosage of Dojolvi is up to 35% of DCI. Patients ranged in age from 7 years to 64 years (median 24 years) and 12 were male. Baseline cardiovascular function in both groups was normal and within test/retest variability normally observed in repeated echocardiograms. After 4 months, patients in both groups had similar mean changes from baseline in left ventricular ejection fraction and wall mass on resting echocardiogram and similar maximal heart rates on treadmill ergometry. Five patients experienced 7 events of rhabdomyolysis in the triheptanoin group and 4 patients experienced 7 events of rhabdomyolysis in the trioctanoin group. No differences were observed between triheptanoin and trioctanoin groups in blood markers of metabolism including glucose, insulin, lactate, total serum, ketones, acylcarnitines, and serum-free fatty acid concentrations.
Adverse effects associated with the use of Dojolvi may include, but are not limited to, the following:
- abdominal pain
The Dojolvi drug label comes with the following warnings and precautions: Feeding Tube Dysfunction: Regularly monitor the tube to ensure proper functioning and integrity. Intestinal Malabsorption in Patients with Pancreatic Insufficiency: Low or absent pancreatic enzymes may reduce absorption of Dojolvi. Avoid administration of Dojolvi in patients with pancreatic insufficiency.
Mechanism of Action
Dojolvi (triheptanoin) is a medium-chain triglyceride consisting of three odd-chain 7-carbon length fatty acids (heptanoate) that provide a source of calories and fatty acids to bypass the long-chain FAOD enzyme deficiencies for energy production and replacement.
For additional information regarding Dojolvi or molecularly confirmed long-chain fatty acid oxidation disorders, please visit the Dojolvy drug label.