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A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

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Trial Profile

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 12 Aug 2024

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms Study 041
  • Sponsors PTC Therapeutics

    • Most Recent Events

    • 01 Jul 2024 According to PTC Therapeutics media release, the company has resubmitted the NDA for Translarna for the treatment of nmDMD in July 2024.
    • 25 Apr 2024 According to PTC Therapeutics media release, Based on FDA feedback, PTC plans to resubmit the NDA for Translarna for the treatment of nmDMD in mid-2024.
    • 25 Jan 2024 According to a PTC Therapeutics, Inc. media release, today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarn (ataluren), which will result in the withdrawal of a therapy for patients in Europe. Per European regulations, the European Commission has 67 days to adopt the opinion.

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