Triheptanoin is under clinical development by Ultragenyx Pharmaceutical and currently in Phase II for Medium-Chain Acyl-CoA Dehydrogenase Deficiency. According to GlobalData, Phase II drugs for Medium-Chain Acyl-CoA Dehydrogenase Deficiency does not have sufficient historical data to build an indication benchmark PTSR for Phase II. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Triheptanoin LoA Report. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Triheptanoin overview

Triheptanoin (Dojolvi) is a triglyceride. It is formulated as liquid for oral route of administration. Dojolvi is indicated for the treatment of pediatric and adult patients with molecularly confirmed long chain fatty acid oxidation disorders.

Triheptanoin is under development for the treatment of long-chain 3-hydroxy acyl-CoA dehydrogenase deficiency, pyruvate dehydrogenase complex (PDC) deficiency, carnitine palmitoyltransferase II deficiency, carnitine-acylcarnitine translocase deficiency and medium-chain acyl-CoA dehydrogenase deficiency (MCADD). The drug candidate is administered orally as a liquid. It is a synthetic triglyceride of heptanoate which is composed of three seven-carbon fatty acids.

It was also under development for the treatment of Pompe disease and sporadic inclusion body myositis (s-IBM). It was under development for Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) patients experiencing disabling paroxysmal movement disorders, Barth syndrome, amyotrophic lateral sclerosis, Rett syndrome, Huntington's disease, Mc Ardle disease (glycogen storage disease type V), glycogen storage disorder type 1, tarui disease, debrancher deficiency and glycogenin-1 deficiency, childhood hemiplegia, migraine, mitochondrial dysfunction in Rett syndrome, dyskinesia and epilepsy. 

Ultragenyx Pharmaceutical overview

Ultragenyx Pharmaceutical (Ultragenyx) is a biotechnology company that develops novel medicines for rare and ultra-rare genetic diseases. It has four marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH); Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

For a complete picture of Triheptanoin’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.