Triheptanoin

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).

Triheptanoin was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2006, 2008, 2014, and 2015.{{cite web | title=Triheptanoin Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=220306 | date=26 May 2006 | access-date=30 June 2020}}{{cite web | title=Triheptanoin Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=220406 | date=1 February 2008 | access-date=30 June 2020}}{{cite web | title=Triheptanoin Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=426414 | date=21 October 2014 | access-date=30 June 2020}}{{dead link|date=May 2025|bot=medic}}{{cbignore|bot=medic}}{{cite web | title=Triheptanoin Orphan Drug Designations and Approvals | website=U.S. Food and Drug Administration (FDA) | url=https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=474015 | date=15 April 2015 | access-date=30 June 2020}}{{dead link|date=May 2025|bot=medic}}{{cbignore|bot=medic}} Triheptanoin was also designated an orphan drug by the European Medicines Agency (EMA).{{cite web | title=EU/3/12/1081 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121081 | access-date=30 June 2020}}{{cite web | title=EU/3/12/1082 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121082 | access-date=30 June 2020}}{{cite web | title=EU/3/15/1495 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151495 | access-date=30 June 2020}}{{cite web | title=EU/3/15/1508 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151508 | access-date=30 June 2020}}{{cite web | title=EU/3/15/1524 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151524 | access-date=30 June 2020}}{{cite web | title=EU/3/15/1525 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151525 | access-date=30 June 2020}}{{cite web | title=EU/3/15/1526 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151526 | access-date=30 June 2020}}{{cite web | title=EU/3/16/1710 | website=European Medicines Agency (EMA) | date=17 September 2018 | url=https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3161710 | access-date=30 June 2020}}

Triheptanoin was approved for medical use in the United States in June 2020.{{cite web | title=Dojolvi: FDA-Approved Drugs | website=U.S. Food and Drug Administration (FDA) | url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=213687 | access-date=30 June 2020}}{{cite press release | title=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 | publisher=Ultragenyx Pharmaceutical | via=GlobeNewswire | date=30 June 2020 | url=https://www.globenewswire.com/news-release/2020/06/30/2055767/0/en/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.html | access-date=30 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.

Triheptanoin is the international nonproprietary name.{{cite journal | vauthors=((World Health Organization)) | year=2019 | title=International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82 | journal=WHO Drug Information | volume=33 | issue=3 | page=694 | hdl=10665/330879 | hdl-access=free | id=License: CC BY-NC-SA 3.0 IGO }}

• Glycerol phenylbutyrate

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• {{cite journal |vauthors=de Almeida Rabello Oliveira M, da Rocha Ataíde T, de Oliveira SL, de Melo Lucena AL, de Lira CE, Soares AA, de Almeida CB, Ximenes-da-Silva A | display-authors=6 |title=Effects of short-term and long-term treatment with medium- and long-chain triglycerides ketogenic diet on cortical spreading depression in young rats |journal=Neurosci. Lett. |volume=434 |issue=1 |pages=66–70 |date=March 2008 |pmid=18281154 |doi=10.1016/j.neulet.2008.01.032 | s2cid=7754768 }}
• {{cite journal | vauthors=Mochel F, DeLonlay P, Touati G, Brunengraber H, Kinman RP, Rabier D, Roe CR, Saudubray JM | display-authors=6 | title=Pyruvate carboxylase deficiency: clinical and biochemical response to anaplerotic diet therapy |journal=Mol. Genet. Metab. | volume=84 | issue=4 | pages=305–12 | date=April 2005 | pmid=15781190 | doi=10.1016/j.ymgme.2004.09.007 }}
• {{cite journal |vauthors=Borges K, Sonnewald U |title=Triheptanoin--a medium chain triglyceride with odd chain fatty acids: a new anaplerotic anticonvulsant treatment? |journal=Epilepsy Res. |volume=100 |issue=3 |pages=239–44 |date=July 2012 |pmid=21855298 |pmc=3422680 |doi=10.1016/j.eplepsyres.2011.05.023 }}

• {{ClinicalTrialsGov|NCT01379625|Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)}}

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Category:Orphan drugs

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