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: 05 Mar 2018

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

    • 20 Feb 2018 According to a PTC Therapeutics media release, the company has received formal dispute resolution request decision from the FDA's Office of New Drugs reiterating the FDA's prior position and denying company's appeal of the CRL in relation to the NDA for ataluren.
    • 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.
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