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Dojolvi

(Triheptanoin)

ApprovedSmall molecule

Triheptanoin is used to treat certain rare genetic disorders that affect how the body breaks down fats for energy, helping to prevent muscle weakness and other serious complications in patients who cannot properly use their normal fat metabolism.[2] Dojolvi is currently approved and in use across 13 indications[4].[1]

Dojolvi is one of 161 small molecule programs targeting Glycogen Storage Disease V. It is currently in Approved.

13

Indications

27

Total Trials

2

Active Trials

160

Competitors

1

FDA Application

NDA/BLA/ANDA regulatory filings

2

Patents

Pipeline

Status by Indication[4][1]

Indication

Glycogen Storage Disease VApproved

Medium Chain Acyl-Coa Dehydrogenase DeficiencyActive, not recruiting

Migraine DisorderApproved

Rett SyndromeActive, not recruiting

Huntington DiseaseApproved

Encephalopathy Due to GLUT1 DeficiencyApproved

Amyotrophic Lateral SclerosisActive, not recruiting

Alternating Hemiplegia of ChildhoodActive, not recruiting

MigraineActive, not recruiting

Medium-Chain Acyl-Coa Dehydrogenase DeficiencyWithdrawn

Glycogen Storage Disease VApproved

Approved

Medium Chain Acyl-Coa Dehydrogenase DeficiencyPhase 2

Active, not recruiting

Migraine DisorderApproved

Approved

Rett SyndromePhase 2

Active, not recruiting

Huntington DiseaseApproved

Approved

Encephalopathy Due to GLUT1 DeficiencyApproved

Approved

Amyotrophic Lateral SclerosisPhase 1

Active, not recruiting

Alternating Hemiplegia of ChildhoodPhase 2

Active, not recruiting

MigrainePhase 1

Active, not recruiting

Medium-Chain Acyl-Coa Dehydrogenase DeficiencyPhase 2

Withdrawn

Showing 10 of 13 indications

Competition

Competitive Landscape

357 competitors in Migraine

#DrugCompanyPhaseTrials

Dojolvi

Aimovig

Emgality

Vyepti

Savella

Cervidil

Botox

RRevance Therapeutics, Inc.

Adempas

Viagra

VViatris Specialty LLC

Pristiq

PPrism Pharmaceuticals

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2027

Phase 3Active, not recruiting

Pediatric Patients

NCT05933200

Q2 2029

Phase 1Recruiting

Children

NCT06340685

Clinical

Trials by Indication[1]

Treatment7 trials

Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Completed·—·Phase 2·NCT00947960·23 enrolled

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Completed·UUniversity of Texas Southwestern Medical Center·Phase 2·NCT03181399·45 enrolled

Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects

Withdrawn·MMaria Luz Couce Pico·Phase 3·NCT02201368

Show 4 more trials

Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)

Completed·JJuan Pascual·Phase 1·NCT02018315·14 enrolled

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

No longer available·—··NCT02968953

Treatment Development of Triheptanoin (G1D)

Completed·JJuan Pascual·Phase 1·NCT03041363·12 enrolled

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder

Completed·OOregon Health and Science University·Phase 2·NCT01379625·32 enrolled

Glycogen Storage Disease2 trials

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Completed·AAreeg El-Gharbawy·Phase 1·NCT03665636·4 enrolled

The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)

Completed·RRigshospitalet, Denmark·Phase 2·NCT02432768·22 enrolled

Medium-chain Acyl-CoA Dehydrogenase Deficiency2 trials

A Phase II, Escalating Dose, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Withdrawn·JJerry Vockley, MD, PhD·Phase 2·NCT06067802

A Phase II, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Withdrawn·JJerry Vockley, MD, PhD·Phase 2·NCT07097311

Pilot Study1 trial

Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome

Unknown status·UUniversity of British Columbia·Phase 2·NCT02000960·3 enrolled

MPA1 trial

Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

Completed·UUniversity of Texas Southwestern Medical Center·Phase 2·NCT03301532·10 enrolled

11 more indications

Migraine1 trial

A Pilot Trial of Triheptanoin for the Preventive Treatment of Migraine

Unknown status·UUniversity of Liege·Phase 1·NCT02784847·10 enrolled

ESS1 trial

Expanded Access to Triheptanoin

Available· Ultragenyx logo

Ultragenyx··NCT03773770

Children1 trial

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Recruiting·JJirair Krikor Bedoyan·Phase 1·NCT06340685·6 enrolled

Rett syndrome1 trial

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

Unknown status·CCenter for Rare Neurological Diseases, Norcross, GA·Phase 2·NCT02696044·12 enrolled

Tolerance1 trial

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Completed·RRigshospitalet, Denmark·Phase 2·NCT03642860·3 enrolled

Metabolism1 trial

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

No longer available·—··NCT01461304

Alternating Hemiplegia of Childhood1 trial

Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood

Completed·IInstitut National de la Santé Et de la Recherche Médicale, France·Phase 2·NCT02408354·10 enrolled

Trial1 trial

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

Completed·AAdrian Lacy·Phase 2·NCT02036853·20 enrolled

Amyotrophic Lateral Sclerosis1 trial

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

Completed·RRichard Bedlack, M.D., Ph.D.·Phase 1/2·NCT03506425·15 enrolled

Pediatric Patients1 trial

A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Active, not recruiting· Ultragenyx logo

Ultragenyx·Phase 3·NCT05933200·69 enrolled

Unspecified4 trials

Triheptanoin in Mc Ardle

Completed·IInstitut National de la Santé Et de la Recherche Médicale, France·Phase 2·NCT02919631·12 enrolled

Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

No longer available·IIrina A Anselm··NCT02500082

Post Study Continuation of C7 for G1D

No longer available·JJuan Pascual··NCT02018302

Show 1 more trial

Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)

Withdrawn·JJuan Pascual·Phase 1/2·NCT02021526

Regulatory

FDA Approval Timeline[5]

Jun 30, 2020

DOJOLVI

NDA NDA213687

Sources

References

[1]
ClinicalTrials.gov — 27 trial records for Triheptanoin. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Dojolvi drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Triheptanoin compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Triheptanoin. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA213687). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Dojolvi. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Ultragenyx company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Ultragenyx

Patent Landscape[6]

Patent Cliff

Apr 28, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

8697748Apr 28, 2029

12551461Nov 14, 2034

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