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Triheptanoin

Chemical compound

Field	Value
type
image	Triheptanoin.svg
image_class	skin-invert-image
width	200
alt	Skeletal formula of triheptanoin
image2	Triheptanoin 3D spacefill.png
image_class2	bg-transparent
width2	240
alt2	Space-filling model of the triheptanoin molecule
pronounce

tradename	Dojolvi
Drugs.com
licence_CA
licence_EU
DailyMedID	Triheptanoin
licence_US
pregnancy_AU
routes_of_administration	By mouth
class	Glycerolipids
ATC_prefix	A16
ATC_suffix	AX17
legal_AU
legal_BR
legal_CA	Rx-only
legal_CA_comment
legal_DE
legal_NZ
legal_UK
legal_US	Rx-only
legal_US_comment
legal_UN
legal_status
CAS_number_Ref
CAS_number	620-67-7
PubChem	69286
DrugBank	DB11677
ChemSpiderID_Ref
ChemSpiderID	62497
UNII_Ref
UNII	2P6O7CFW5K
KEGG	D11465
ChEMBL	4297585
synonyms	UX007
IUPAC_name	2,3-di(heptanoyloxy)propyl heptanoate or glyceryl triheptanoate
C	24	H = 44	O = 6
SMILES	CCCCCCC(=O)OCC(COC(=O)CCCCCC)OC(=O)CCCCCC
StdInChI_Ref
StdInChI	1S/C24H44O6/c1-4-7-10-13-16-22(25)28-19-21(30-24(27)18-15-12-9-6-3)20-29-23(26)17-14-11-8-5-2/h21H,4-20H2,1-3H3
StdInChIKey_Ref
StdInChIKey	PJHKBYALYHRYSK-UHFFFAOYSA-N

| Drugs.com =

| elimination_half-life =

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).

The most common adverse reactions include abdominal pain, diarrhea, vomiting, and nausea.

Triheptanoin was approved for medical use in the United States in June 2020.

Triheptanoin is a triglyceride that is composed of three seven-carbon (C7:0) fatty acids. These odd-carbon fatty acids are able to provide anaplerotic substrates for the TCA cycle. Triheptanoin is used clinically in humans to treat inherited metabolic diseases, such as pyruvate carboxylase deficiency and carnitine palmitoyltransferase II deficiency. It also appears to increase the efficacy of the ketogenic diet as a treatment for epilepsy.

Since triheptanoin is composed of odd-carbon fatty acids, it can produce ketone bodies with five carbon atoms, as opposed to even-carbon fatty acids which are metabolized to ketone bodies with four carbon atoms. The five-carbon ketones produced from triheptanoin are beta-ketopentanoate and beta-hydroxypentanoate. Each of these ketone bodies easily crosses the blood–brain barrier and enters the brain.

Medical uses

Dojolvi is indicated as a source of calories and fatty acids for the treatment of children and adults with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

History

Triheptanoin was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2006, 2008, 2014, and 2015. Triheptanoin was also designated an orphan drug by the European Medicines Agency (EMA).

Triheptanoin was approved for medical use in the United States in June 2020.

The FDA approved triheptanoin based on evidence from three clinical trials (Trial 1/NCT018863, Trial 2/NCT022141 and Trial 3/NCT01379625). The trials enrolled children and adults with LC-FAOD. Trials 1 and 2 were conducted at 11 sites in the United States and the United Kingdom, and Trial 3 was conducted at two sites in the United States.

Trial 1 and Trial 2 were used to evaluate the side effects of triheptanoin. Both trials enrolled children and adults diagnosed with LC-FAOD. In Trial 1, participants received triheptanoin for 78 weeks. Trial 2 enrolled participants from other trials who were already treated with triheptanoin (including those from Trial 1) as well as participants who were never treated with triheptanoin before. Trial 2 is still ongoing and is planned to last up to five years.

The benefit of triheptanoin was evaluated in Trial 3 which enrolled children and adults with LC-FAOD. Half of the participants received triheptanoin and half received trioctanoin for four months. Neither the participants nor the investigators knew which treatment was given until the end of the trial. The benefit of triheptanoin in comparison to trioctanoin was assessed by measuring the changes in heart and muscle function.

Names

Triheptanoin is the international nonproprietary name.

References

(23 October 2014). "Summary Basis of Decision (SBD) for Dojolvi".
(30 June 2020). "Dojolvi- triheptanoin liquid".
(May 2025). "Drug Trials Snapshots: Dojolvi".
(26 May 2006). "Triheptanoin Orphan Drug Designations and Approvals".
(1 February 2008). "Triheptanoin Orphan Drug Designations and Approvals".
(May 2025). "Triheptanoin Orphan Drug Designations and Approvals".
(May 2025). "Triheptanoin Orphan Drug Designations and Approvals".
(17 September 2018). "EU/3/12/1081".
(17 September 2018). "EU/3/12/1082".
(17 September 2018). "EU/3/15/1495".
(17 September 2018). "EU/3/15/1508".
(17 September 2018). "EU/3/15/1524".
(17 September 2018). "EU/3/15/1525".
(17 September 2018). "EU/3/15/1526".
(17 September 2018). "EU/3/16/1710".
"Dojolvi: FDA-Approved Drugs".
(30 June 2020). "Ultragenyx Announces U.S. FDA Approval of Dojolvi (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders". Ultragenyx Pharmaceutical.
(2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82". WHO Drug Information.

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This article was imported from Wikipedia and is available under the Creative Commons Attribution-ShareAlike 4.0 License. Content has been adapted to SurfDoc format. Original contributors can be found on the article history page.

orphan-drugs triglycerides

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