PTC whiffs on another Duchenne study, plans to go to FDA again anyway
Once again PTC Therapeutics’ Duchenne muscular dystrophy drug has failed a clinical trial. And once again Stuart Peltz and his crew are trying to use the data to prove why the FDA should approve it.
PTC announced late Thursday that in its latest Phase II study, their flagship drug Translarna failed to significantly improve dystrophin expression across 20 patients when measured by either of the assays used for the trial. The trial, known as Study 045, joins larger Phase IIb and Phase III Duchenne studies and one Phase III cystic fibrosis trial on the list of pivotal failures for a drug that first entered the clinic nearly two decades ago and has thrice been batted down by the FDA.
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