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A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis

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Trial Profile

A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 12 Apr 2022

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Cystic fibrosis
  • Focus Registrational; Therapeutic Use
  • Sponsors PTC Therapeutics

    • Most Recent Events

    • 06 Mar 2017 According to an European Medicines Agency media release dated March 2017, PTC Therapeutics International Limited officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for Translarna to be used to treat cystic fibrosis. This decision was based on the data from this trial which failed to show that Translarna was more effective than placebo at improving lung function or reducing flare-ups in the overall study population.
    • 10 Jun 2016 New analyses from this trial were presented at the 39th European Cystic Fibrosis Conference, according to a PTC Therapeutics media release.
    • 30 Sep 2015 According to a PTC Therapeutics media release, the EMA has validated the submission of a variation for a new indication for ataluren [Translarna] for the treatment of nonsense mutation cystic fibrosis for patients not taking chronic inhaled aminoglycoside antibiotics.

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