PTC discusses FDA's reasons for Translarna rejection

PTC Therapeutics Inc. (NASDAQ:PTCT) said FDA found that data from two clinical trials included in an NDA for Translarna ataluren to treat Duchenne muscular dystrophy (DMD) "were negative and do not provide substantial evidence of effectiveness." Last week, PTC received a refuse to file letter from FDA for the NDA (see BioCentury Extra, Feb. 23).

According to PTC, FDA said the company's adjustments to its Phase III ACT DMD study of Translarna were post hoc and therefore did not show efficacy. The NDA also included data from a Phase IIb study. ...