A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy
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
- 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.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History