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‘File Over Protest’ At US FDA: PTC To Pursue Rarely-Used Pathway For DMD Drug

 
• By Michael Cipriano

Translarna’s best path forward is the file over protest pathway for the application, which received a refuse to file letter last year, PTC says; former OND Director John Jenkins calls it ‘a fairly rare event.’

In another unusual twist for US regulatory review of Duchenne muscular dystrophy products, PTC Therapeutics Inc.will send its European Union-approved Translarna (ataluren) through the rarely-used File Over Protest process at FDA.

"Feedback indicated this process, rather than continued appeal, is the best path forward for the current Translarna NDA to receive a full and fair review," the company said in a...

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EMA Says Digital Transformation A ‘Fundamental Shift’ In Meeting Regulatory Challenges

 
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New EU Approvals

 
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EMA Says Digital Transformation A ‘Fundamental Shift’ In Meeting Regulatory Challenges

 
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EMA: ‘Sad To See’ Member States Against Patient Engagement Under EU Pharma Reform

 
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The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.