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Expedited Review Pathways: Three More Breakthroughs, Third RMAT Awarded

 
• By Bridget Silverman

US FDA's popular breakthrough therapy designation given to Proteon's vonapanitase for dialysis patients, Synthetic Biologics' ribaxamase to prevent C. difficile infection, and River Vision's teprotumumab for thyroid eye disease; Vericel's cell therapy for heart failure is designated a regenerative medicine advanced therapy.

Bacterias 3D rendering

The latest breakthrough therapy designation (BTD) awards from FDA illustrate the expedited review program's appeal for sponsors who are entering Phase III in areas where there is little relevant precedent to guide trial design.

Increased communication with US regulators will help advanceSynthetic Biologics Inc.'s microbiome therapeutic, Proteon Therapeutics Inc.'s recombinant vasodilator, and...

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More from Review Pathways

US FDA’s Rising First-Cycle Complete Response Rate Draws Congress’ Attention

 
• By Sue Sutter

The House Appropriations Committee wants an analysis of how issues that led to CRLs could have been resolved within the first review cycle and seeks an agency crackdown on counterfeit GLP-1 agonists.

What UK’s Clinical Trials Overhaul Means For Orphan Drug Developers

 
• By Eliza Slawther

The UK government has acknowledged that sponsors of clinical trials for rare diseases face challenges around patient recruitment and trial design, which will be addressed in its clinical trials reform, a lawyer says.

Nipah Vaccine With ‘Pandemic Potential’ Wins EMA PRIME Designation

 
• By Neena Brizmohun

The developers of the ChAdOx1 NipahB vaccine will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.

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.

More from Pathways & Standards

Nipah Vaccine With ‘Pandemic Potential’ Wins EMA PRIME Designation

 
• By Neena Brizmohun

The developers of the ChAdOx1 NipahB vaccine will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.

Elegy For SACHRP: US HHS Research Protection Panel Tackled Tough Pediatric Trial Questions

 
• By Bridget Silverman

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

House Gives US FDA More Non-User Fee Funds Than Requested

 
• By Sue Sutter and Derrick Gingery

A US House bill would give the FDA $33.1m more in budget authority than requested by the Trump Administration for fiscal year 2026. The measure was sent to the full House Appropriations committee on a party-line vote.