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A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

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Trial Profile

A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

Status: Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 30 Jan 2026

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At a glance

  • Drugs Vatiquinone (Primary)
  • Indications Friedreich's ataxia
  • Focus Registrational; Therapeutic Use
  • Acronyms MOVE-FA
  • Sponsors PTC Therapeutics

Most Recent Events

  • 12 Jan 2026 According to a PTC Therapeutics media release, FDA requested additional information from MOVE-FA Phase 3 trial prior to providing guidance on next steps
  • 27 Feb 2025 According to a PTC Therapeutics media release, Vatiquinone for children and adults with Friedreich's ataxia, granted priority review with a target regulatory action date of August 19, 2025.
  • 19 Feb 2025 According to a PTC Therapeutics media release, the company announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreichs ataxia (FA) based on data from the placebo-controlled MOVE-FA study

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