New Drug Approvals

Home » 2020 APPROVALS » Triheptanoin




Read all about Organic Spectroscopy on ORGANIC SPECTROSCOPY INTERNATIONAL 


Blog Stats

  • 4,242,600 hits

Enter your email address to follow this blog and receive notifications of new posts by email.

Join 2,802 other subscribers

add to any


Skeletal formula of triheptanoin


Approved US FDA 30/6/2020 Dojolvi UX 007

Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation.4 In clinical trials, patients with long chain fatty acid oxidation disorders (lc-FAODs) treated with triheptanoin are less likely to develop hypoglycemia, cardiomyopathy, rhabdomyolysis, and hepatomegaly.1,2 Complications in lc-FAOD patients are reduced from approximately 60% to approximately 10% with the addition of triheptanoin.2

Triheptanoin was granted FDA approval on 30 June 2020.4

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).[1][2][3]

The most common adverse reactions include abdominal pain, diarrhea, vomiting, and nausea.[1][2][3]

Triheptanoin was approved for medical use in the United States in June 2020.[4][2][3]

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).[1][2]


Triheptanoin was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2006, 2008, 2014, and 2015.[5][6][7][8] Triheptanoin was also designated an orphan drug by the European Medicines Agency (EMA).[9][10][11][12][13][14][15][16]

Triheptanoin was approved for medical use in the United States in June 2020.[4][2]

The FDA approved triheptanoin based on evidence from three clinical trials (Trial 1/NCT018863, Trial 2/NCT022141 and Trial 3/NCT01379625).[3] The trials enrolled children and adults with LC-FAOD.[3] 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.[3]

Trial 1 and Trial 2 were used to evaluate the side effects of triheptanoin.[3] Both trials enrolled children and adults diagnosed with LC-FAOD.[3] In Trial 1, participants received triheptanoin for 78 weeks.[3] 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.[3] Trial 2 is still ongoing and is planned to last up to five years.[3]

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


Triheptanoin is the international nonproprietary name.[17]


Synthesis of triheptanoin and formulation as a solid diet for rodents -  Semak - 2012 - European Journal of Lipid Science and Technology - Wiley  Online Library


  1. Jump up to:a b c d “Dojolvi- triheptanoin liquid”DailyMed. 30 June 2020. Retrieved 24 September2020.
  2. Jump up to: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”. Ultragenyx Pharmaceutical. 30 June 2020. Retrieved 30 June 2020 – via GlobeNewswire.
  3. Jump up to: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.
  4. Jump up to:a b “Dojolvi: FDA-Approved Drugs”U.S. Food and Drug Administration (FDA). Retrieved 30 June 2020.
  5. ^ “Triheptanoin Orphan Drug Designations and Approvals”U.S. Food and Drug Administration (FDA). 26 May 2006. Retrieved 30 June 2020.
  6. ^ “Triheptanoin Orphan Drug Designations and Approvals”U.S. Food and Drug Administration (FDA). 1 February 2008. Retrieved 30 June 2020.
  7. ^ “Triheptanoin Orphan Drug Designations and Approvals”U.S. Food and Drug Administration (FDA). 21 October 2014. Retrieved 30 June 2020.
  8. ^ “Triheptanoin Orphan Drug Designations and Approvals”U.S. Food and Drug Administration (FDA). 15 April 2015. Retrieved 30 June 2020.
  9. ^ “EU/3/12/1081”European Medicines Agency (EMA). Retrieved 30 June 2020.
  10. ^ “EU/3/12/1082”European Medicines Agency (EMA). Retrieved 30 June 2020.
  11. ^ “EU/3/15/1495”European Medicines Agency (EMA). Retrieved 30 June 2020.
  12. ^ “EU/3/15/1508”European Medicines Agency (EMA). Retrieved 30 June 2020.
  13. ^ “EU/3/15/1524”European Medicines Agency (EMA). Retrieved 30 June 2020.
  14. ^ “EU/3/15/1525”European Medicines Agency (EMA). Retrieved 30 June 2020.
  15. ^ “EU/3/15/1526”European Medicines Agency (EMA). Retrieved 30 June 2020.
  16. ^ “EU/3/16/1710”European Medicines Agency (EMA). Retrieved 30 June 2020.
  17. ^ World Health Organization (2019). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82”. WHO Drug Information33 (3): 694. hdl:10665/330879. License: CC BY-NC-SA 3.0 IGO.

Further reading

External links

  • “Triheptanoin”Drug Information Portal. U.S. National Library of Medicine.
  • Clinical trial number NCT01379625 for “Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)” at
Clinical data
Trade namesDojolvi
Other namesUX007
AHFS/Drugs.comProfessional Drug Facts
License dataUS DailyMedTriheptanoin
US: N (Not classified yet)
Routes of
By mouth
Drug classGlycerolipids
ATC codeNone
Legal status
Legal statusUS: ℞-only [1]
IUPAC name[show]
CAS Number620-67-7 
PubChem CID69286
CompTox Dashboard (EPA)DTXSID40862306 
ECHA InfoCard100.009.681 
Chemical and physical data
Molar mass428.610 g·mol−1
3D model (JSmol)Interactive image
InChI[hide]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, Dojolvi,  UX 007, FDA 2020, 2020 APPROVALS

Prescription Products

DojolviLiquid0.96 g/1mLOralUltragenyx Pharmaceutical Inc.2020-07-01Not applicableUS flag

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

This site uses Akismet to reduce spam. Learn how your comment data is processed.


Follow New Drug Approvals on

Enter your email address to follow this blog and receive notifications of new posts by email.

Join 2,802 other subscribers


DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with AFRICURE PHARMA, ROW2TECH, NIPER-G, CLEANCHEM LABS as ADVISOR, earlier assignment was with GLENMARK LIFE SCIENCES LTD, as CONSUlTANT, Retired from GLENMARK in Jan2022 Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 32 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 32 PLUS year tenure till date Feb 2023, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 100 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 100 Lakh plus views on dozen plus blogs, 227 countries, 7 continents, He makes himself available to all, contact him on +91 9323115463, email, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 38 lakh plus views on New Drug Approvals Blog in 227 countries...... , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc He has total of 32 International and Indian awards

Personal Links

View Full Profile →



Follow my blog with Bloglovin The title of your home page You could put your verification ID in a comment Or, in its own meta tag Or, as one of your keywords Your content is here. The verification ID will NOT be detected if you put it here.
%d bloggers like this: