PTC sinks on stalled Translarna appeal, CHMP setback

PTC Therapeutics Inc. (NASDAQ:PTCT) fell $5.30 (40%) to $7.97 on Monday after it said FDA denied its first appeal of the refuse-to-file letter the agency issued for Translarna ataluren to treat 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" (see BioCentury Extra, Feb. 29). ...