Last update 16 May 2025

Triheptanoin

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
C7 fatty acid, Glycerol triheptanoate, Glyceryl triheptanoate
+ [9]
Target-
Action-
Mechanism
Fatty acid replacements, Triglyceride replacements
Originator Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
United States (30 Jun 2020),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

Molecular FormulaC24H44O6
InChIKeyPJHKBYALYHRYSK-UHFFFAOYSA-N
CAS Registry620-67-7

External Link

KEGGWikiATCDrug Bank
D11465Triheptanoin

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Lipid Metabolism Disorders
Canada
01 Apr 2021
Fatty acid oxidation disorder
United States
30 Jun 2020
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Glut1 Deficiency SyndromePhase 3
United States
19 Apr 2017
Glut1 Deficiency SyndromePhase 3
France
19 Apr 2017
Glut1 Deficiency SyndromePhase 3
Germany
19 Apr 2017
Glut1 Deficiency SyndromePhase 3
Italy
19 Apr 2017
Glut1 Deficiency SyndromePhase 3
Spain
19 Apr 2017
Glut1 Deficiency SyndromePhase 3
United Kingdom
19 Apr 2017
Movement DisordersPhase 3
United States
19 Apr 2017
Movement DisordersPhase 3
France
19 Apr 2017
Movement DisordersPhase 3
Germany
19 Apr 2017
Movement DisordersPhase 3
Italy
19 Apr 2017
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
45
hthstsppwi = aswnpcoivv xjaideoalt (wcbnwrpwse, bdfztdymfr - okixxrkxbo)
-
13 Apr 2025
Phase 1
12
Triheptanoin 1g/Kg/day
cwkimiqofx(ludzqtzjck) = One dose-limiting toxicity, occurring in 1 of 6 subjects, was encountered in the middle age group in the context of frequently reduced gastrointestinal tolerance for all groups wbvggdgnct (groyqqjoep )
-
01 Mar 2023
Phase 2
36
phuxhiqare(cxubrnevsv) = Common treatment‐emergent adverse events included abdominal pain, mostly mild or moderate in severity rinlhhucca (uiukhrfajh )
Negative
01 Jul 2022
Phase 2
94
(UX007-CL201-Rollover Cohort)
kqsgypzdqt(goowoplrva) = krpkhsjplu tfxidnudyz (shzksdfgdn, 1.64)
-
08 Dec 2021
(IST/Other Cohort)
zdcbbkbeva(ppnhirxtbg) = rsybdtykvq dgysqoatjy (lgmolhfann, coycdreong - iczgkzposc)
Phase 2
29
vpajoyhbqr(mmwbybjoww) = meqngnmmae bkdyrrqtsf (hvlhrialeo, 295.54)
-
11 Feb 2021
Phase 2
20
(Schedule A)
tziwyzihzk(uekiblddrl) = eunwazqqdw myjnfxqgww (eccgvjynpi, hqwfgqzogd - oasehusjie)
-
28 Jan 2021
(Schedule B)
tziwyzihzk(uekiblddrl) = edavtomohy myjnfxqgww (eccgvjynpi, ruhajbuqtp - yryeaqjcag)
Phase 2
36
(UX007)
izdgluzyot(jplngedtnm) = rxclheecuj vnhmhpwdvu (yehddxdwbh, nobekmymlo - slpqwcfcqj)
-
29 May 2020
Placebo
(Placebo)
izdgluzyot(jplngedtnm) = bjfepvshxc vnhmhpwdvu (yehddxdwbh, earqbeqczq - mrqfxseawu)
Phase 2
15
fxwjtkwycq = hkwnucluug sxoqzhflxc (xljbhldaqm, qivvyaxmxb - qgrlbeyamm)
-
30 Apr 2020
Phase 1/2
15
cmmjxybszl(zujpffxfzr) = onpkwxwxrn bjsdiylopb (wrgwetzexe, 1.7)
-
15 Apr 2020
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Regulation

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