CHMP rebuff's PTC's ataluren for DMD
EMA's CHMP recommended against conditional approval of Translarna ataluren from PTC Therapeutics Inc. (NASDAQ:PTCT) to treat nonsense mutation Duchenne muscular dystrophy (DMD). CHMP said ataluren failed to show improvement in six-minute walking distance (6MWD) over placebo after 48 weeks of treatment and that data on other efficacy measures related to a patient's daily activities provided "limited support" of ataluren's benefits. The MAA includes efficacy data from a single 174-patient Phase IIb trial. Additionally, CHMP said PTC did not provide sufficient data on ataluren's mechanism of action in the body and its effects with changing doses. PTC said it plans to request a reexamination. Top-line data from a confirmatory Phase III trial of ataluren -- a small molecule that facilitates complete translation of proteins containing nonsense mutations -- in DMD are expected in mid-2015. ...