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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...

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UK MHRA: Bacteriophage Developers Wanted ‘More Clarity’ On UK Framework

 
• By Eliza Slawther

Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.

User Fee-Funded Staff Would Drop In FY 2026 US FDA Budget

 
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EMA Official Clarifies Benefits And Limitations Of Innovation Pathways

 
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US FDA’s Prasad: ‘We Will Always Embrace Surrogate Endpoints’

 
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The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.

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MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
• By Eliza Slawther

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.

US FDA Cell-Gene Therapy Head Says Agency Has Revived Stalled Programs

 
• By Sarah Karlin-Smith

CBER's Nicole Verdun wants rare disease sponsors with stalled treatments to circle back because the FDA's evolved thinking on clinical trial designs may offer another opportunity for cast away products.

FDA’s FY 2026 Budget Request Lacks New Policy Proposals

 
• By Sue Sutter

The White House requested $6.8bn for the FDA, down 3.9% from the current funding level, but does not propose any legislative changes. In previous years, the agency used the budget process to seek statutory fixes specific to generic drugs and shortages.