Triheptanoin

Triheptanoin
Clinical data
Pronunciation	/traɪˈhɛptənɔɪn/; try-HEP-tə-noyn
Trade names	Dojolvi
Other names	UX007
AHFS/Drugs.com	Professional Drug Facts
License data	US DailyMed: Triheptanoin;
Routes of; administration	By mouth
Drug class	Glycerolipids
ATC code	A16AX17 (WHO) ;
Legal status
Legal status	CA: ℞-only; US: ℞-only;
Identifiers
	IUPAC name 2,3-di(heptanoyloxy)propyl heptanoate or glyceryl triheptanoate;
CAS Number	620-67-7 ;
PubChem CID	69286;
DrugBank	DB11677;
ChemSpider	62497 ;
UNII	2P6O7CFW5K;
KEGG	D11465;
ChEMBL	ChEMBL4297585;
CompTox Dashboard (EPA)	DTXSID40862306 ;
ECHA InfoCard	100.009.681
Chemical and physical data
Formula	C24H44O6
Molar mass	428.610 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CCCCCCC(=O)OCC(COC(=O)CCCCCC)OC(=O)CCCCCC;
	InChI InChI=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 ; Key:PJHKBYALYHRYSK-UHFFFAOYSA-N ;

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).^[2]^[3]^[4]

The most common adverse reactions include abdominal pain, diarrhea, vomiting, and nausea.^[2]^[3]^[4]

Triheptanoin was approved for medical use in the United States in June 2020.^[5]^[3]^[4]

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.

^ "Summary Basis of Decision (SBD) for Dojolvi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
^ ^a ^b ^c "Dojolvi- triheptanoin liquid". DailyMed. 30 June 2020. Retrieved 24 September 2020.
^ ^a ^b ^c Cite error: The named reference Ultragenyx PR was invoked but never defined (see the help page).
^ ^a ^b ^c "Drug Trials Snapshots: Dojolvi". U.S. Food and Drug Administration. 30 June 2020. Retrieved 16 July 2020.
^ Cite error: The named reference FDA Dojolvi was invoked but never defined (see the help page).

[1] "Summary Basis of Decision (SBD) for Dojolvi". Health Canada. 23 October 2014. Retrieved 29 May 2022.

[Dojolvi_FDA_label-2] "Dojolvi- triheptanoin liquid". DailyMed. 30 June 2020. Retrieved 24 September 2020.

[Ultragenyx_PR-3] Cite error: The named reference Ultragenyx PR was invoked but never defined (see the help page).

[FDA_snapshot-4] "Drug Trials Snapshots: Dojolvi". U.S. Food and Drug Administration. 30 June 2020. Retrieved 16 July 2020.

[FDA_Dojolvi-5] Cite error: The named reference FDA Dojolvi was invoked but never defined (see the help page).

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