Triheptanoin

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.

• 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). DOJOLVI is a medium-chain triglyceride 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).

• None. None.contraindicated

Assess metabolic requirements by determining daily caloric intake (DCI) prior to calculating the dose of DOJOLVI. ( 2.1 ) For patients receiving another medium-chain triglyceride product, discontinue prior to the first dose of DOJOLVI. ( 2.3 ) 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 mixed thoroughly into semi-solid food/liquid or medical food/formula at mealtimes or with snacks. ( 2.2 ) See the full prescribing information for instructions on how to calculate the volume per dose; initiate and titrate the dosage to achieve the target; and prepare and administer DOJOLVI. ( 2.2 , 2.3 , 2.4 ) 2.1 Important Recommendations Prior to DOJOLVI Treatment 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. 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. 2.2 Recommended Dosage 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 by mixing thoroughly into semi-solid food/liquid or medical food/formula at mealtimes or with snacks. In order to reach a target daily dosage, patients may require an increase in their total fat intake.

Feeding Tube Dysfunction : Regularly monitor the feeding tube to ensure proper functioning and integrity. ( 5.1 ) 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. ( 5.2 ) 5.1 Feeding Tube Dysfunction Feeding tube performance and functionality can degrade over time depending on usage and environmental conditions. In clinical trials, feeding tube dysfunction was reported in patients receiving triheptanoin. The contribution of DOJOLVI cannot be ruled out. Do not administer DOJOLVI in feeding tubes manufactured of polyvinyl chloride (PVC) [see Dosage and Administration (2.4 ] . Regularly monitor the feeding tube to ensure proper functioning and integrity. 5.2 Intestinal Malabsorption in Patients with Pancreatic Insufficiency Pancreatic enzymes hydrolyze triheptanoin and release heptanoate as medium-chain fatty acids in the small intestine. Low or absent pancreatic enzymes may result in reduced absorption of heptanoate subsequently leading to insufficient supplementation of medium-chain fatty acids. Avoid administration of DOJOLVI in patients with pancreatic insufficiency.

Most common adverse reactions are (≥10%): abdominal pain, diarrhea, vomiting, and nausea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety population included 79 patients with LC-FAOD exposed to DOJOLVI in two studies: one open-label 78-week study of DOJOLVI in 29 patients (Study 1) followed by an open-label extension study (Study 2). Twenty-four patients from Study 1 continued into Study 2. Patients ranged from 4 months to 63 years of age and the population was 52% male. Of the 79 patients, 87% were White, 5% were Black or African-American, 4% were Asian, and 4% other. The daily dosage of DOJOLVI ranged between 12% and 41% DCI (which corresponds to 0.7 g/kg/day to 6.0 g/kg/day for pediatric patients and 0.5 g/kg/day to 1.3 g/kg/day for adult patients) for a mean duration of 23 months. The most common adverse reactions to DOJOLVI reported in the pooled safety population of Study 1 and Study 2 were gastrointestinal (GI)-related, and included abdominal pain (abdominal discomfort, abdominal distension, abdominal pain, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].

8.1 Pregnancy Risk Summary There are no available data on triheptanoin use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies conducted in pregnant rats and rabbits administered triheptanoin during the period of organogenesis, the primary toxicological effect (reduced body weight gain) was considered to be specific to decreased food consumption related to taste aversion in animals, and therefore is not relevant to clinical use in the intended populations. There is a pregnancy safety study for DOJOLVI. If a patient becomes pregnant while receiving DOJOLVI, healthcare providers should report DOJOLVI exposure by calling Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.

Metabolism

Following oral administration, triheptanoin is extensively hydrolyzed to heptanoate and glycerol by pancreatic lipases in the intestines. The exposure of triheptanoin in the human plasma is minimal.

• Jun 30, 2020NDANDA213687Ultragenyx Pharm Inc

1. 1Vomiting8216%
2. 2Diarrhoea7114%
3. 3Rhabdomyolysis5410%
4. 4Off Label Use509.6%
5. 5Abdominal Pain Upper407.7%
6. 6Blood Creatine Phosphokinase Increased356.7%
7. 7Abdominal Discomfort346.5%
8. 8Death326.2%
9. 9Nausea275.2%
10. 10Gastrointestinal Disorder244.6%
11. 11Hospitalisation244.6%
12. 12Illness234.4%
13. 13Abdominal Pain203.8%
14. 14Metabolic Disorder183.5%
15. 15Product Dose Omission Issue163.1%

The 10 most recently updated of 40 ClinicalTrials.gov registrations naming Triheptanoin as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
  Completed · Phase 2 · Interventional · 10 enrolled · Institut National de la Santé Et de la Recherche Médicale, France
  NCT01882062updated 2026-06-11
• Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
  Active not recruiting · Observational · 150 enrolled · Ultragenyx Pharmaceutical Inc
  NCT04632953updated 2026-06-03
• A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
  Completed · Phase 2 · Interventional · 100 enrolled · Institut National de la Santé Et de la Recherche Médicale, France
  NCT02453061updated 2026-06-03
• Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
  Suspended · Phase 2 · Interventional · 8 enrolled · Jerry Vockley, MD, PhD
  NCT06067802updated 2026-04-21
• Expanded Access to Triheptanoin
  Available · Expanded access · Ultragenyx Pharmaceutical Inc
  NCT03773770updated 2026-04-13
• A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
  Active not recruiting · Phase 3 · Interventional · 69 enrolled · Ultragenyx Pharmaceutical Inc
  NCT05933200updated 2026-04-07
• Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
  Recruiting · Phase 2 · Interventional · 24 enrolled · Jerry Vockley, MD, PhD
  NCT07097311updated 2026-03-05
• Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
  Recruiting · Phase 1 · Interventional · 6 enrolled · Jirair Krikor Bedoyan
  NCT06340685updated 2026-02-17
• Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood
  Completed · Phase 2 · Interventional · 10 enrolled · Institut National de la Santé Et de la Recherche Médicale, France
  NCT02408354updated 2025-12-10
• Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS
  Completed · Phase 2 · Interventional · 20 enrolled · Institut National de la Santé Et de la Recherche Médicale, France
  NCT02014883updated 2025-10-03

Frequently asked questions

How does Triheptanoin work?

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.

What is Triheptanoin used for?

According to FDA labeling, Triheptanoin carries indications including: 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). DOJOLVI is a medium-chain triglyceride 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).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Triheptanoin?

Triheptanoin is classified as Various alimentary tract and metabolism products, Medium-chain Triglyceride.

What are the brand names for Triheptanoin?

Triheptanoin is marketed under brand names including Dojolvi.

What are the contraindications for Triheptanoin?

Triheptanoin labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.