Rolling submission to FDA for Translarna (ataluren) completed for Duchenne muscular dystrophy indication- PTC Therapeutics

PTC Therapeutics has completed its rolling submission of a New Drug Application (NDA) to the FDA for Translarna (ataluren), an oral therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC has also fulfilled the principal requirement of the European Medicines Agency (EMA) in connection with its approval of Translarna in August 2014 by submitting the results of its Phase III ACT DMD clinical trial to the agency.

Results from the Phase III ACT DMD trial showed clinically meaningful benefits for Translarna-treated patients. In the overall intent-to-treat study population, the primary endpoint of change from baseline in the 6-minute walk test (6MWT) demonstrated a 15 metre benefit, which was not statistically significant. A highly significant benefit of 47 meters was demonstrated in the pre-specified patient population of 300-400 metres at baseline as measured by the 6MWT, which is in line with the Company's prior experience in its Phase IIb trial and consistent with the evolving understanding of the 6MWT. Importantly, no patients in this group lost ambulation versus four patients in the placebo group.