Drug Profile

Vatiquinone -PTC Therapeutics

Alternative Names: Alpha-tocotrienol quinone - PTC Therapeutics; EPI-743; PTC 743; Vincerenone

Latest Information Update: 15 Sep 2025

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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

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Epilepsy; Friedreich's ataxia; Leigh disease
New Molecular Entity Yes

Preregistration Friedreich's ataxia
Phase III 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

08 Sep 2025 PTC Therapeutics in collaboration with Medical University of South Carolina initiates an expanded access programme for Mitochondrial disorders (In adults, In adolescents In children, In infants, Treatment-experienced) (NCT07159139)
19 Aug 2025 PTC Therapeutics receives complete response letter from the FDA for Vatiquinone in Friedreich's ataxia
15 Apr 2025 PTC Therapeutics completes a phase-III clinical trials in Mitochondrial disorders (In children, In adults, In the elderly) in the USA, France, Italy, Japan, Poland, United Kingdom, Spain, Sweden (PO) (NCT05218655)

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