PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has commenced a rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial.
"The initiation of our NDA submission for Translarna marks another significant milestone towards providing Translarna to all nonsense mutation Duchenne muscular dystrophy patients," stated Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We look forward to the completion of the ACT DMD confirmatory Phase 3 clinical trial so that we can finalize the NDA. Gaining US approval, in addition to Translarna's European approval, will help to make Translarna available to patients across the globe. This is our commitment to the patients, families, advocacy groups and physicians who have worked and supported PTC Therapeutics through many years of research and development."
"We want Translarna available for Duchenne patients in the US as fast as possible," stated Pat Furlong, President and Founder of PPMD. "We commend PTC's perseverance and dedicated efforts to speed access to Translarna for people with Duchenne in the United States. Every day counts for people with Duchenne. Our hope is that PTC's efforts pave the way for Translarna as well as other therapies in the US."
SOURCE PTC Therapeutics, Inc.