Drug Profile
Vatiquinone -PTC Therapeutics
Alternative Names: Alpha-tocotrienol quinone - PTC Therapeutics; EPI-743; PTC 743; VincerenoneLatest Information Update: 05 Mar 2024
Price :
$50
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At a glance
- Originator Edison Pharmaceuticals
- Developer PTC Therapeutics; Sumitomo Pharma; University of Florida; Yale University
- Class Alkadienes; Antiepileptic drugs; Antiparkinsonians; Benzoquinones; Cyclohexenes; Small molecules
- Mechanism of Action 15-lipoxygenase inhibitors; Antioxidants; NQO1 modulators
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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
- Phase III Friedreich's ataxia; Mitochondrial disorders
- Phase II/III Epilepsy
- Phase II Leigh disease; Methylmalonic acidaemia; Noise-induced hearing loss; Parkinson's disease; Rett syndrome
- No development reported Gilles de la Tourette's syndrome
Most Recent Events
- 29 Feb 2024 PTC Therapeutics completes Type C meeting with the US FDA for Vatiquinone NDA submission in Friedreich ataxia
- 29 Feb 2024 PTC Therapeutics plans to submit NDA to US FDA for Friedreich's ataxia in the late 2024
- 08 Jan 2024 PTC Therapeutics expects meeting with US FDA for to support an NDA submission in Friedreich's ataxia