TheraRadar
Data updated: May 26, 2026

DOJOLVI (triheptanoin)

Trial Activity: Declining 3 active trials
Orphan Drug Fast Track
Rare Disease Approved 2020-06-30

DOJOLVI is indicated for the treatment of Long-Chain Fatty Acid Oxidation Disorders.

Source: FDA Label • ULTRAGENYX PHARM INC • Medium-chain Triglyceride

How DOJOLVI Works

Triheptanoin is a medium-chain triglyceride consisting of three odd-chain, 7-carbon length fatty acids known as heptanoate. These fatty acids provide a source of calories and lipids that bypass the specific enzyme deficiencies associated with long-chain fatty acid oxidation disorders. By circumventing these metabolic blocks, the drug enables the body to produce energy and replace necessary fatty acids.

Development Insights

Juan Pascual conducting 3 trials (18%)
25 indications explored (Broad Platform)
glucose transporter type 1 deficiency syndrome (4 trials)
glut1ds1 (3 trials)
medium-chain acyl-coa dehydrogenase deficiency (2 trials)
1
Indication
--
Phase 3 Trials
5
Years on Market

Details

Status
Prescription
First Approved
2020-06-30
Patent Cliff
2034

Pro Metrics

Patent cliff and revenue data

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Routes
ORAL
Dosage Forms
LIQUID

Companies

Active Ingredient: TRIHEPTANOIN

DOJOLVI Approval History

2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
2 FDA actions from 2020 to 2023
Oct 2023 SUPPL
Label · Labeling
Jun 2020 ORIGINAL
New Drug · Type 1 - New Molecular Entity

What DOJOLVI Treats

1 indications

DOJOLVI is approved for 1 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Long-Chain Fatty Acid Oxidation Disorders
Source: FDA Label

DOJOLVI Competitive Set

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Three rings of competition based on shared molecular targets and treated indications.

Indication competitors

1

Same indication, different mechanism — what else might this patient receive?

Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

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Clinical Trial Registry

17 trials
Trial Sponsor ID Phase Status Title
NCT06067802 STUDY23040121 UX007-IST237 Ph 2 suspended Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
NCT05933200 UX007-CL302 2022-001539-10, 2023-509809-76-00 Ph 3 active not recruiting A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
NCT07097311 STUDY25050173 Ph 2 recruiting Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
NCT06340685 STUDY23050042 UX007-IST236 Ph 1 recruiting Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
NCT02408354 HEMIHEP C14-53 Ph 2 completed Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood
NCT02919631 C15-54 Ph 2 completed Triheptanoin in Mc Ardle
NCT03181399 results posted 122016-013 1R01NS094257-01A1 Ph 2 completed Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
NCT03642860 #20171012 Trihep 2017-004153-17 Ph 2 completed The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses
NCT03301532 results posted STU 102015-091 R01NS094257-01A1 Ph 2 completed Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
NCT03041363 STU 062016-105 R01NS094257 Ph 1 completed Treatment Development of Triheptanoin (G1D)
NCT02036853 GLUT1DS results posted 2013-NEUR-001 Ph 2 completed An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome
NCT03506425 results posted Pro00092250 Ph 1, Ph 2 completed A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
NCT02018315 results posted UTSW 122010-186 Ph 1 completed Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
NCT02432768 H-8-2014-006 2014-003644-12 Ph 2 completed The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)
NCT02021526 PASCG1D2014 Ph 1, Ph 2 withdrawn Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)
NCT00947960 009-103 Ph 2 completed Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
NCT01379625 Triheptanoin results posted FD003895 Ph 2 completed Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
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Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

DOJOLVI FDA Label Details

Indications & Usage

FDA Label (PDF)

DOJOLVI is indicated for the treatment of Long-Chain Fatty Acid Oxidation Disorders.

View full patent landscape →
1 OB patents · 1 families · 25 international docs across 5 countries

DOJOLVI Patents & Exclusivity

Latest Patent: Nov 2034
Exclusivity: Jun 2027

Patents (2 active)

US12551461 Expires Nov 14, 2034
US8697748 Expires Apr 28, 2029

Exclusivity

ODE-311 Until Jun 2027
Source: FDA Orange Book

Pro Intelligence Preview

Deep insights for DOJOLVI

Revenue Insights

  • Quarterly revenue tracking
  • Historical trend analysis

Patent Timeline

  • Cliff: 2034
  • 2 active patents

Trial Analysis

  • 17 total trials
  • Stage: Declining

Competitive Landscape

  • Competitor tracking
  • Same target/indication analysis
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Full approval history • All patents • Revenue trends • Competitor analysis

Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.

How We Calculate These Metrics

Trial Activity Stage

Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.

Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.

  • Growth: High proportion of early-phase trials (Phase 1/2), active development
  • Expansion: Significant Phase 3 activity, approaching or pursuing approvals
  • Mature: Substantial Phase 4 post-marketing studies
  • Stable: Mixed phase distribution, steady development
  • Declining: Low active trial ratio, reduced R&D investment