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triheptanoin

Trade name: Dojolvi

Small moleculeapprovedOrphan Drug FDAFast Track FDA

Approved

Jun 30, 2020

Triheptanoin, sold under the brand name Dojolvi, is a medication for the treatment of children and adults with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). — Wikipedia

Clinical trial activity

39 trials · 24 clinical orgs · 1 marketing orgs

Phase 1
10
Phase 2
27
Phase 3
3
Phase 4
0

Earliest trial started Jun 1, 2006 (NCT00328159)

Timeline

2000s

  1. Jan 1, 2009

    Earliest Phase 2 Sponsor(trial)

  2. Jan 1, 2009

    Earliest Phase 3 Sponsor(trial)

2010s

  1. Jan 1, 2012

    Earliest Phase 1 Sponsor(trial)

  2. Apr 15, 2015

    Orphan Drug Designation

2020s

  1. Jun 30, 2020

    Ultragenyx Inc. — Earliest FDA Approval

  2. Jun 30, 2020

    Ultragenyx Inc. — Marketing Organization

  3. Jun 30, 2020

    Ultragenyx Inc. — NDA Secondary Org

  4. Jun 30, 2020

    Ultragenyx Inc. — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

  • Fatty acidsACTIVATOR

    Fatty acids activator

  • Unspecified targetOTHER

    Supplement

    Source of calories and fatty acids in long-chain FAOD enzyme deficiencies.

Approval history

  • approvedFast trackJun 30, 2020

Chemistry & pharmacology

Loading structure…

SMILES

CCCCCCC(=O)OCC(COC(=O)CCCCCC)OC(=O)CCCCCC
Mol. weight
428.61 g/mol
Lipinski Ro5
1 violation(s)
Rule of 3
No
Chirality
Achiral Molecule
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

Sources

Also known as

  • glyceroltriheptanoate
  • glyceryl triheptanoate
  • triheptanoin
  • triheptanoin
  • triheptanoin
  • triheptanoin
  • triheptanoin oil

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07097311Phase 2Nov 30, 2025Ultragenyx Inc., University of Pittsburgh
NCT06340685Phase 1Jul 11, 2024Ultragenyx Inc., University of Pittsburgh
NCT06067802Phase 2Dec 31, 2023Ultragenyx Inc., University of Pittsburgh
NCT05933200Phase 3Feb 28, 2023Ultragenyx Inc.
NCT04513002Phase 2Mar 31, 2021National Health and Medical Research Council, Australia, Queensland Children's Hospital, University of Queensland
NCT03665636Early Phase 1 (Phase 1)Sep 7, 2018Ultragenyx Inc., University of Pittsburgh
NCT03642860Phase 2Aug 15, 2018Ultragenyx Inc., University of Copenhagen
NCT03301532Early Phase 1 (Phase 1)Jul 1, 2018National Institute of Neurological Disorders and Stroke (NINDS), The University of Texas, Dallas
NCT03506425Phase 1/Phase 2 (Phase 2)Jun 21, 2018Duke University, Ultragenyx Inc.
NCT03181399Phase 2Jun 30, 2017National Institute of Neurological Disorders and Stroke (NINDS), The University of Texas, Dallas
NCT03041363Phase 1Mar 29, 2017National Institute of Neurological Disorders and Stroke (NINDS), The University of Texas, Dallas
NCT02960217Phase 3Jan 31, 2017Ultragenyx Inc.
NCT02968953N/ANov 1, 2016Ultragenyx Inc., University of Pittsburgh
NCT02919631Phase 2Oct 1, 2016Institut National de la Santé Et de la Recherche Médicale, France, University of Copenhagen
NCT02784847Early Phase 1 (Phase 1)Jun 1, 2016University of Liege
NCT02696044Phase 2Jun 1, 2016Emory University, Ultragenyx Inc.
NCT02679235Phase 1/Phase 2 (Phase 2)Apr 1, 2016Ultragenyx Inc., University of Sherbrooke
NCT02599961Phase 2Sep 1, 2015Ultragenyx Inc.
NCT02500082N/AJul 14, 2015Harvard University
NCT02453061Phase 2Jun 1, 2015Institut National de la Santé Et de la Recherche Médicale, France, Ultragenyx Inc.
Showing 20 of 39 trials
Page 1 / 2

Organizations

Research & Development (24)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Ultragenyx Inc.For profit22742009
The University of Texas, DallasAcademic/Hospital8732012
University of PittsburghAcademic/Hospital7632009
Institut National de la Santé Et de la Recherche Médicale, FranceAcademic/Hospital5512013
National Institute of Neurological Disorders and Stroke (NINDS)Government3022017
University of CopenhagenAcademic/Hospital3212015
University of ParisAcademic/Hospital2122006
Baylor UniversityAcademic/Hospital1112009
Cook Children's Medical CenterAcademic/Hospital1112014
Duke UniversityAcademic/Hospital1112018
Emory UniversityAcademic/Hospital1112016
Harvard UniversityAcademic/Hospital1112015
National Health and Medical Research Council, AustraliaGovernment1012021
Oregon Health and Science UniversityAcademic/Hospital1112011
OsakidetzaAcademic/Hospital1012009
Queensland Children's HospitalAcademic/Hospital1012021
Universidad Complutense de MadridAcademic/Hospital1012009
Universitario de Santiago, SpainAcademic/Hospital1112009
University of British ColumbiaAcademic/Hospital1112014
University of LiegeAcademic/Hospital1112016
24 organizations
Page 1 / 2

Marketing (3)

OrganizationOrg typeRelationshipDate
Ultragenyx Inc.For profitMKTGJun 30, 2020
Ultragenyx Inc.For profitNDA2Jun 30, 2020
Ultragenyx Inc.For profitNDAJun 30, 2020