Triheptanoin - Ultragenyx Pharmaceutical
Alternative Names: C7 fatty acid; C7 oil; Dojolvi; Glycerol triheptanoate; Glyceryl triheptanoate; UX-007Latest Information Update: 04 Mar 2024
At a glance
- Originator Baylor Research Institute
- Developer Ultragenyx Pharmaceutical
- Class Esters; Small molecules; Triglycerides
- Mechanism of Action Triglyceride replacements
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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
- 31 Dec 2023 Ultragenyx Pharmaceutical has patent protection for triheptanoin in USA, Australia, Brazil, Canada, Israel, Korea, Malaysia, and Taiwan
- 27 Dec 2022 Phase-III clinical trials in Lipid metabolism disorders (In adolescents, In children, In infants, In neonates) in Spain, Poland, Czech Republic (PO) (EudraCT2022-001539-10)
- 31 Dec 2021 Launched for Lipid metabolism disorders (In adolescents, In children, In infants, In adults) in Canada (PO) prior to December 2021