dm+d

Unassigned

New Medicines

DojolviLong-chain fatty acid oxidation disorders

Information

Dojolvi
New molecular entity
Ultragenyx
Ultragenyx

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Launched
Yes
Yes
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].

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 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

Glucose transporter type 1 (GLUT 1) deficiency syndrome (de Vivo disease)

Information

New molecular entity
Ultragenyx
Ultragenyx

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Yes
Yes
Oct 19No longer listed in Ultragenyx pipeline for Glut1 DS; assume development discontinued in light of PIII study being terminated [10].

Category

Synthetic medium-chain triglyceride
Glut 1 deficiency syndrome is a rare disease with an estimated birth incidence of 1:90,000 [3]. There are currently around 500 cases diagnosed worldwide [4].
Glucose transporter type 1 (GLUT 1) deficiency syndrome (de Vivo disease)
Oral

Trial or other data

Oct 19PIII trial (NCT02960217) has been halted prematurely due to lack of efficacy [9].