Translarna ataluren regulatory update

PTC said FDA denied the company’s first appeal of the refusal to file letter the agency issued for Translarna ataluren to treat nonsense mutation Duchenne muscular dystrophy (DMD). The company said it plans to escalate its appeal via “multiple cycles” that go to “progressively higher levels” at FDA.

FDA issued the letter in February. At the time, PTC said the agency found that Translarna’s NDA submission failed to show “substantial evidence of effectiveness.” PTC said the compound should be given the same opportunity for full review given to other recent DMD applicants, including an advisory committee meeting. Last month, FDA granted accelerated approval to DMD treatment Exondys 51 eteplirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT, Cambridge, Mass.) despite concerns voiced at an April advisory committee meeting (see BioCentury, Sept. 26)...