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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: 05 Nov 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

  • 30 Oct 2024 According to PTC Therapeutics media release, the U.S. FDA has accepted for review the resubmission of the NDA for Translarna for the treatment of nmDMD based on the data from this and Study 041 studies respectively.
  • 28 Jun 2024 According to a PTC Therapeutics, media release, the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nmDMD. This opinion follows the return of the previously issued negative opinion by the EC for re-review. PTC plans to request re-examination of the opinion. The marketing authorization for Translarna remains in effect, pending the outcome of the re-examination procedure and subsequent EC ratification.
  • 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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