The company said that the results of the trial were not statistically significant, in which the primary endpoint of change from baseline in the six-minute walk test (6MWT) showed a 15m benefit, in the overall intent-to-treat study population.
PTC said that significant benefit of 47m was showed in the pre-specified patient population of 300-400m at baseline as measured by the 6MWT, which is in line with the firm’s prior experience in the Phase 2b trial.
PTC Therapeutics CEO Stuart Peltz said: "The totality of the data from our two robust placebo-controlled studies across over 400 patients demonstrate a clinically relevant impact on patients’ lives.
"We plan to submit these results to the EMA and complete our NDA submission to the FDA by the end of the year."
Both placebo and treated patients have been given the chance to continue on Translarna in an open-label extension study, after the completion of ACT DMD. Around 97% of the patients who completed ACT DMD enrolled in the extension study.
ACT DMD is a placebo-controlled multi-center, randomized and double-blind trial, which included around 228 patients in 53 sites across 18 countries.
The study included patients between the age group seven and 16 with nmDMD and were randomized to receive either Translarna 40mg/kg per day or placebo over 48 weeks.
Important secondary outcome measures in the study were timed-function tests, including time to run or walk 10m and the time to ascend or descend four stairs.
The tertiary endpoints comprised of the North Star ambulatory assessment test, a functional scale designed for ambulant boys affected by DMD, and the pediatric outcomes data collection instrument (PODCI), a validated tool for measuring quality of life in pediatric patients with orthopedic conditions.