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Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

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Trial Profile

Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

Status: Active, no longer recruiting
Phase of Trial: Phase IV

Latest Information Update: 01 Feb 2024

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions
  • Acronyms STRIDE
  • 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.
    • 05 Dec 2023 According to a PTC Therapeutics media release, the company has submitted the briefing document as part of the re-examination of the initial Committee for Medicinal Products for Human Use (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna in Europe. This procedure will focus only on the renewal of the existing conditional marketing authorization and expected to last until late January 2024, when an opinion will be provided.
    • 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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