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Vatiquinone -PTC Therapeutics

Drug Profile

Vatiquinone -PTC Therapeutics

Alternative Names: EPI-743; PTC 743; Vincerenone

Latest Information Update: 25 May 2021

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At a glance

  • Originator Edison Pharmaceuticals
  • Developer PTC Therapeutics; Sumitomo Dainippon Pharma; University of Florida; Yale University
  • Class Alkadienes; Antiepileptic drugs; Antiparkinsonians; Benzoquinones; Cyclohexenes; Small molecules
  • Mechanism of Action Antioxidants; NQO1 modulators
  • Orphan Drug Status

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

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

Highest Development Phases

  • Phase III Friedreich's ataxia
  • 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

  • 13 May 2021 Discontinued - Phase-III for Leigh disease in Japan (PO)
  • 30 Nov 2020 Phase-III clinical trials in Friedreich's ataxia (In children, In adults, In the elderly) in USA (PO, Capsule) (NCT04577352)
  • 29 Oct 2020 PTC therapeutics plans a registrational phase III MOVE-FA trial for Friedreich Ataxia in the US, Australia, Canada and in Italy Q4 2020 (NCT04577352)
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