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Vatiquinone -PTC Therapeutics

Drug Profile

Vatiquinone -PTC Therapeutics

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

Latest Information Update: 05 Mar 2024

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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 - Leigh disease; Epilepsy; Friedreich's ataxia
  • 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
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