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A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Trial Profile

A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 01 Feb 2024

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Registrational; Therapeutic Use
  • 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.
    • 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.
    • 15 Sep 2023 According to a PTC Therapeutics media release, company announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy on the basis of Study 041, Studies 007 and 020. Company plans to submit a request for re-examination per EMA guidelines.
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