NDAORALLIQUID
Approved
Jun 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
6/8 — Strong
DOJOLVI (triheptanoin) by Ultragenyx Pharmaceutical is 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. First approved in 2020.
Drug data last refreshed 15h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
DOJOLVI (triheptanoin) is an oral liquid medium-chain triglyceride approved in June 2020 for treating long-chain fatty acid oxidation disorders (LCFAOD). It provides a caloric and fatty acid source that bypasses deficient long-chain FAO enzymes, enabling patients to produce energy through alternative metabolic pathways.
$0.003BPart D
Peak8.4 years to LOEProduct is at peak lifecycle stage with stable niche market penetration in orphan indication; team size remains modest but strategically positioned within rare disease portfolio.
Mechanism of Action
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.
Pharmacologic Class:
Medium-chain Triglyceride
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Started Aug 2026
8 enrolled
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Started Jul 2026
24 enrolled
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
Started Jul 2024
6 enrolled
Pyruvate Dehydrogenase Complex Deficiency
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Started Feb 2023
69 enrolled
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
Started Oct 2020
4 enrolled
Glycogen Storage Disease Type I
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.