Translarna regulatory update

PTC said FDA acknowledged receipt of the biotech’s NDA for Translarna ataluren to treat nonsense mutation Duchenne muscular dystrophy (nmDMD), which the company submitted over protest. The agency granted standard review to the NDA, with an Oct. 24 PDUFA date. In February 2016, PTC said FDA issued a refusal to file letter for Translarna, citing data that "do not provide substantial evidence of effectiveness," as well as a lack of data addressing potential abuse of the therapy (see BioCentury, Feb. 29, 2016)...