Once More Unto The Breach: PTC's Duchenne Drug Translarna Gets Advisory Cmte.
US FDA's Peripheral and Central Nervous System Drugs Advisory Committee to consider application, which PTC Therapeutics filed over protest.
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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.
Several members of the Peripheral and Central Nervous System Drugs Advisory Committee meeting looking at PTC' Therapeutics' proposed Duchenne muscular dystrophy treatment also looked at Sarepta's Exondys 51 and BioMarin's Kyndrisa, but most did not vote or comment favorably.
Recent and upcoming US FDA advisory committee meetings and a summary of topics covered.