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How Accelerated Approval Works – And How It Doesn't

 
• By Derrick Gingery

PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.

FDA Advisory Committee Feature image

US FDA officials are refusing to allow PTC Therapeutics Inc.'s Translarna (ataluren) to use the precedent for accelerated approval in Duchenne muscular dystrophy that was created by the controversial approval of Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen).

Agency officials told the Peripheral and Central Nervous System Drugs Advisory Committee Sept. 28 that because the efficacy studies submitted were negative or problematic, the pathway could not be used.

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