for nonsense mutation Duchenne muscular dystrophy
PTC Therapeutics withdrew its NDA resubmission for Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy on 2026-02-12. Resubmission had followed prior FDA rejections; the company elected to withdraw rather than pursue further FDA review.
Duchenne muscular dystrophy is a severe, inherited muscle-wasting disease that leads to progressive weakness and loss of mobility. Translarna (ataluren) is designed to help muscle cells ignore certain genetic "stop" signals, allowing them to produce a functional dystrophin protein. This approach aims to address the underlying cause of the disease and could potentially slow its progression.
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after i...
Source: ClinicalTrials.gov