Trial Profile
A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
Status:
Completed
Phase of Trial:
Phase III
Latest Information Update: 01 Feb 2024
Price :
$35
*
At a glance
- Drugs Ataluren (Primary)
- Indications Duchenne muscular dystrophy
- Focus Registrational; Therapeutic Use
- Acronyms Study 041
- Sponsors PTC Therapeutics
- 25 Jan 2024 According to a PTC Therapeutics, Inc. media release, today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarn (ataluren), which will result in the withdrawal of a therapy for patients in Europe. Per European regulations, the European Commission has 67 days to adopt the opinion.
- 20 Sep 2023 Status changed from active, no longer recruiting to completed.
- 15 Sep 2023 According to a European Medicines Agency media release, CHMP has recommended not renewing the marketing authorisation for Translarna following the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorization. The committee concluded that Translarna's benefit-risk balance is negative. The review considered results from studies, data from a patient registry and the views of consulted experts in neurology and patient representatives.