PTC jumps on CHMP reversal for ataluren

PTC Therapeutics Inc. (NASDAQ:PTCT) jumped as much as $7.94 (52%) on Friday before closing the day up $4.71 (31%) to $20.03 after EMA's CHMP recommended conditional approval of Translarna ataluren to treat nonsense mutation Duchenne muscular dystrophy (DMD), reversing a prior negative opinion. In January, CHMP recommended against conditional approval and said Translarna failed to show improvement in six-minute walking distance (6MWD) over placebo after 48 weeks of treatment in a 174-patient Phase IIb trial (see BioCentury Extra, Jan. 24).

PTC requested a reexamination and resubmitted an additional analysis of efficacy data from the trial. The company did not respond in time for publication with details about the additional analysis, but said it showed that ataluren led to a "clinically meaningful" 31.3 meter benefit in 6MWD (p=0.0561). PTC previously reported a 29.7 meter improvement (p=0.058). Top-line data from a confirmatory Phase III trial are expected in mid-2015. Translarna is a small molecule that facilitates complete translation of proteins containing nonsense mutations. ...