PTC Therapeutics submits PTC 124 to the EMA for treatment of patients with Duchenne Muscular Dystrophy

A Marketing Authorization Application to the EMA seeking conditional approval for PTC 124 (ataluren), from PTC Therapeutics, for the treatment of patients with nonsense mutation Duchenne Muscular Dystrophy (nmDMD) has been accepted. Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review process.

The submission is based on a 48-week, 174-patient Phase IIb study showing that nmDMD patients treated with ataluren (10, 10, 20 mg/kg given daily) walked on average 31.3 meters farther than patients on placebo, as measured by the change in six-minute walk distance (6MWD) from baseline to Week 48. Patients receiving ataluren demonstrated a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD.