dm+d
Unassigned
New Medicines
Dojolvi
Long-chain fatty acid oxidation disordersInformation
Dojolvi
New molecular entity
Ultragenyx
Ultragenyx
Development and Regulatory status
Phase III Clinical Trials
Phase III Clinical Trials
Launched
Yes
Yes
Oct 20
Ultragenyx is in ongoing discussions with EU regulators [11].
Jul 20
Launched in US as Dojolvi [10].
Jun 20
Approved in US [9].
Oct 19
The 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 18
Still listed as PII in Ultragenyx pipeline [7].
Jan 17
Still listed as PII in Ultragenyx pipeline [5].
Jul 15
EMA 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 15
FDA granted orphan drug designation for triheptanoin for the treatment of fatty acid oxidation disorders (FAOD) [3].
Category
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
Oral
Trial or other data
Aug 16
NCT01886378 study completed; results will be used by Ultragenyx to identify the optimum patient population for a PIII trial [6].
Apr 15
PII (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 14
PII (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 14
A 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].