NDAORALLIQUID
Approved
Jun 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
8/8 — Comprehensive
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. Approved for long-chain fatty acid oxidation disorders. First approved in 2020.
Drug data last refreshed Yesterday · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
DOJOLVI (triheptanoin) is a medium-chain triglyceride oral liquid that bypasses long-chain fatty acid oxidation defects by providing calories and energy through an alternative metabolic pathway. It treats 21 rare metabolic and neurological disorders including long-chain FAOD, glycogen storage diseases, and neurodegenerative conditions like Huntington disease and ALS. The drug works by supplying heptanoate (7-carbon fatty acids) that can be metabolized without requiring the deficient enzymes in patients' energy production pathways.
$3MPart D
Peak8.5 years to LOEProduct is at peak lifecycle with minimal direct competition, suggesting stable but niche commercial operations with a focused field team.
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 (1)
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.