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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

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 21 Jun 2019

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At a glance

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

    • 24 Jul 2018 Post hoc analysis of the placebo arm of this trial were published in Muscle & Nerve, according to a PTC Therapeutics media release.
    • 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.
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