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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: 01 Feb 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

    • 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.
    • 20 Sep 2023 Status changed from active, no longer recruiting to completed.
    • 15 Sep 2023 According to a European Medicines Agency media release, CHMP has recommended not renewing the marketing authorisation for Translarna following the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorization. The committee concluded that Translarna's benefit-risk balance is negative. The review considered results from studies, data from a patient registry and the views of consulted experts in neurology and patient representatives.
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