A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Trial Profile

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Completed
Phase of Trial: Phase III

Latest Information Update: 15 Dec 2017

At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy; Muscular dystrophies
  • Focus Registrational; Therapeutic Use
  • Acronyms ACT DMD
  • Sponsors PTC Therapeutics
  • Most Recent Events

    • 02 Nov 2017 According to a PTC Therapeutics media release, company has filed a formal dispute resolution request challenging FDA's decision of not approving ataluren NDA in its present form.
    • 25 Oct 2017 According to a PTC Therapeutics media release, the Office of Drug Evaluation I of the US FDA has issued a complete response letter (CRL) for the NDA of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies. The letter from the Office of Drug Evaluation I of the FDA stated that it is unable to approve the application in its current form.
    • 18 Jul 2017 According to a PTC Therapeutics media release, results from this trial published in The Lancet.
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