Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review

 
• By Sue Sutter
  
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FDA has twice refused to file PTC Therapeutics' NDA for the Duchenne muscular dystrophy treatment because of 'clearly and convincingly negative' pivotal trials and questionable post hoc analyses; sponsor argues ataluren should be afforded the same consideration and flexibility given to Sarepta's DMD drug eteplirsen, which received a controversial accelerated approval.

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PTC Therapeutics Inc. is counting on the precedent set by the US FDA's controversial Exondys 51 decision, coupled with strong patient community support for its Duchenne muscular dystrophy drug Translarna (ataluren), to overcome two pivotal studies that US FDA staff describe as so "clearly and convincingly negative" they were considered a bar to even reviewing the company's new drug application (NDA).

On Sept. 28, the Peripheral and Central Nervous System Drugs Advisory Committee will discuss PTC's ataluren oral suspension for the...

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