Prnewswire·2026-02-12 22:00Regulatory Update - PTC Therapeutics has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (DMD) due to FDA feedback indicating the data is unlikely to meet the effectiveness threshold for approval [1] - The CEO of PTC Therapeutics expressed disappointment over the inability to achieve FDA approval after over two decades of development efforts for a therapy aimed at boys and young men with nonsense mutation DMD [1] Product Information - Translarna (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation, which halts essential protein synthesis [1] - Duchenne muscular dystrophy is a rare and fatal genetic disorder primarily affecting males, leading to progressive muscle weakness and premature death due to heart and respiratory failure [1] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing clinically differentiated medicines for rare disorders [1] - The company aims to provide access to best-in-class treatments for patients with unmet medical needs through a robust and diversified pipeline of transformative medicines [1]