Triheptanoin is a Small Molecule owned by Ultragenyx Pharmaceutical, and is involved in 28 clinical trials, of which 23 were completed, 4 are ongoing, and 1 is planned.

Triheptanoin is a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD. The drug candidate provides two tricarboxylic acid cycle (TCA) substrates that are converted from fat to energy by the TCA cycle. The substrate replacement therapy may exhibit therapeutic intervention by checking the underlying cause of disease progression.

The revenue for Triheptanoin is expected to reach a total of $4.2bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Triheptanoin NPV Report.

Triheptanoin was originated by Baylor University Medical Center and Case Western Reserve University and is currently owned by Ultragenyx Pharmaceutical.

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 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, long-chain 3-hydroxy acyl-CoA dehydrogenase deficiency, carnitine palmitoyltransferase II deficiency, carnitine-acylcarnitine translocase deficiency, childhood hemiplegia, migraine, mitochondrial dysfunction in Rett syndrome, Barth syndrome, dyskinesia and epilepsy. 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.

Ultragenyx Pharmaceutical Overview

Ultragenyx Pharmaceutical (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three 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). 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.

The company reported revenues of (US Dollars) US$351.4 million for the fiscal year ended December 2021 (FY2021), an increase of 29.7% over FY2020. The operating loss of the company was US$381.7 million in FY2021, compared to an operating loss of US$330.1 million in FY2020. The net loss of the company was US$454 million in FY2021, compared to a net loss of US$186.6 million in FY2020. The company reported revenues of US$90.7 million for the third quarter ended September 2022, an increase of 1.5% over the previous quarter.

Quick View – Triheptanoin

Report Segments
  • Innovator (NME)
Drug Name
  • Triheptanoin
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
  • Metabolic Disorders
  • Musculoskeletal Disorders
Key Companies
Highest Development Stage
  • Marketed

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

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.