Additional Relevant Details
As previously disclosed, PTC recently participated in discussions with the U.S. Food and Drug Administration (FDA) to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of nmDMD. PTC recently submitted an appeal to escalate continuing discussions about the RTF decision to the next level of FDA management via the formal dispute resolution process within FDA's Center for Drug Evaluation and Research (CDER). This process exists to encourage open, prompt discussion of scientific and procedural disputes that arise during the drug development process between FDA and companies. Within the dispute resolution process, PTC is willing to consider multiple paths to advance a potential FDA approval, including the possibility of conducting an additional clinical trial under accelerated approval.