Triheptanoin
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Pronunciation | /traɪˈhɛptənɔɪn/ try-HEP-tə-noyn |
Trade names | Dojolvi |
Other names | UX007 |
AHFS/Drugs.com | Professional Drug Facts |
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Glycerolipids | |
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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
Since triheptanoin is composed of odd-carbon fatty acids, it can produce
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).[2][3]
History
Triheptanoin was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2006, 2008, 2014, and 2015.[6][7][8][9] Triheptanoin was also designated an orphan drug by the European Medicines Agency (EMA).[10][11][12][13][14][15][16][17]
Triheptanoin was approved for medical use in the United States in June 2020.[5][3]
The FDA approved triheptanoin based on evidence from three clinical trials (Trial 1/NCT018863, Trial 2/NCT022141 and Trial 3/NCT01379625).[4] The trials enrolled children and adults with LC-FAOD.[4] 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.[4]
Trial 1 and Trial 2 were used to evaluate the side effects of triheptanoin.[4] Both trials enrolled children and adults diagnosed with LC-FAOD.[4] In Trial 1, participants received triheptanoin for 78 weeks.[4] 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.[4] Trial 2 is still ongoing and is planned to last up to five years.[4]
The benefit of triheptanoin was evaluated in Trial 3 which enrolled children and adults with LC-FAOD.[4] Half of the participants received triheptanoin and half received trioctanoin for four months.[4] Neither the participants nor the investigators knew which treatment was given until the end of the trial.[4] The benefit of triheptanoin in comparison to trioctanoin was assessed by measuring the changes in heart and muscle function.[4]
Names
Triheptanoin is the international nonproprietary name.[18]
See also
References
- ^ "Summary Basis of Decision (SBD) for Dojolvi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
- ^ a b c d "Dojolvi- triheptanoin liquid". DailyMed. 30 June 2020. Retrieved 24 September 2020.
- ^ a b c d e "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" (Press release). Ultragenyx Pharmaceutical. 30 June 2020. Retrieved 30 June 2020 – via GlobeNewswire.
- ^ a b c d e f g h i j k l m n o "Drug Trials Snapshots: Dojolvi". U.S. Food and Drug Administration. 30 June 2020. Retrieved 16 July 2020.
- ^ a b "Dojolvi: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Retrieved 30 June 2020.
- ^ "Triheptanoin Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 26 May 2006. Retrieved 30 June 2020.
- ^ "Triheptanoin Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 1 February 2008. Retrieved 30 June 2020.
- ^ "Triheptanoin Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 21 October 2014. Retrieved 30 June 2020.
- ^ "Triheptanoin Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 15 April 2015. Retrieved 30 June 2020.
- ^ "EU/3/12/1081". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/12/1082". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/15/1495". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/15/1508". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/15/1524". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/15/1525". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/15/1526". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- ^ "EU/3/16/1710". European Medicines Agency (EMA). 17 September 2018. Retrieved 30 June 2020.
- hdl:10665/330879. License: CC BY-NC-SA 3.0 IGO.
Further reading
- de Almeida Rabello Oliveira M, da Rocha Ataíde T, de Oliveira SL, de Melo Lucena AL, de Lira CE, Soares AA, et al. (March 2008). "Effects of short-term and long-term treatment with medium- and long-chain triglycerides ketogenic diet on cortical spreading depression in young rats". Neurosci. Lett. 434 (1): 66–70. S2CID 7754768.
- Mochel F, DeLonlay P, Touati G, Brunengraber H, Kinman RP, Rabier D, et al. (April 2005). "Pyruvate carboxylase deficiency: clinical and biochemical response to anaplerotic diet therapy". Mol. Genet. Metab. 84 (4): 305–12. PMID 15781190.
- Borges K, Sonnewald U (July 2012). "Triheptanoin--a medium chain triglyceride with odd chain fatty acids: a new anaplerotic anticonvulsant treatment?". Epilepsy Res. 100 (3): 239–44. PMID 21855298.
External links
- Clinical trial number NCT01379625 for "Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)" at ClinicalTrials.gov