New Medicines

DojolviLong-chain fatty acid oxidation disorders


New molecular entity

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Oct 20Ultragenyx is in ongoing discussions with EU regulators [11].
Jul 20Launched in US as Dojolvi [10].
Jun 20Approved in US [9].
Oct 19The FDA has accepted for review the NDA for triheptanoin for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy [8].
Jan 18Still listed as PII in Ultragenyx pipeline [7].
Jan 17Still listed as PII in Ultragenyx pipeline [5].
Jul 15EMA Orphan Status for treatment of long-chain 3-hydroxyacyl-coA dehydrogenase deficiency and also treatment of very long-chain acyl-CoA dehydrogenase deficiency [4].
Apr 15FDA granted orphan drug designation for triheptanoin for the treatment of fatty acid oxidation disorders (FAOD) [3].


Synthetic medium-chain triglyceride
The estimated prevalence based on screening for Western populations is 0.67 per 100,000 [1].
Long-chain fatty acid oxidation disorders

Trial or other data

Aug 16NCT01886378 study completed; results will be used by Ultragenyx to identify the optimum patient population for a PIII trial [6].
Apr 15PII (NCT01379626) study to determine if triheptanoin can decrease muscle pain and increase the heart function and amount of energy in patients with LC-FAODs has completed in the US. Study results are not yet available [2].
Dec 14PII (NCT02214160) open-label extension study for subjects previously enrolled in triheptanoin studies in US. The study is designed to obtain long-term safety information and evaluate maintenance of efficacy in a diverse LC-FAOD polulation and will allow patients to be treated with triheptanoin for up to 3 years or until market approval [2].
Feb 14A PII (NCT01886378) open-label study to assess the safety and efficacy of triheptanoin in subjects with LC-FAOD enrolls first patient. The study aims to recruit 30 subjects (>6 months of age); following a 4 week run in period patients will receive treatment for 24 weeks, followed by an additional 54 week extension period. The estimated primary completion date is Aug 15 [2].

Evidence based evaluations