PTC Therapeutics provides regulatory update on Translarna (ataluren) for the treatment of DMD

PTC Therapeutics Inc. announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following  FDA feedback on the application review.

“FDA shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency’s threshold of substantial evidence of effectiveness to support approval of Translarna. We have therefore made the decision to withdraw the NDA submission,” said Dr. Matthew B. Klein, Chief Executive Officer of PTC Therapeutics. “We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and are disappointed that FDA approval cannot be achieved.”