Triheptanoin - Ultragenyx Pharmaceutical
Alternative Names: C7 fatty acid; C7 oil; Dojolvi; Glycerol triheptanoate; Glyceryl triheptanoate; UX-007Latest Information Update: 07 Nov 2024
At a glance
- Originator Baylor Research Institute
- Developer Ultragenyx Pharmaceutical
- Class Esters; Small molecules; Triglycerides
- Mechanism of Action Triglyceride replacements
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Marketed Lipid metabolism disorders
- Phase II Glycogen storage disease; Glycogen storage disease type V; Hemiplegia; Huntington's disease; Rett syndrome
- Phase I/II Amyotrophic lateral sclerosis
- No development reported Glycogen storage disease type I
- Discontinued De Vivo disease
Most Recent Events
- 05 Nov 2024 Ultragenyx Pharmaceutical announces intention to submit NDA to PMDA for Lipid metabolism disorders in mid-2025
- 05 Nov 2024 Ultragenyx Pharmaceutical receives positive finalized assessment report with agreement to file for Conditional Early Approval (CEA) from the PMDA for triheptanoin in lipid metabolism disorders prior to November 2024
- 31 Dec 2023 Ultragenyx Pharmaceutical has patent protection for triheptanoin in USA, Australia, Brazil, Canada, Israel, Korea, Malaysia, and Taiwan