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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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National Priority Voucher Gives US FDA Commissioner Tool For Radical Regulatory Surgery

 
• By Bridget Silverman

US FDA Commissioner Martin Makary will establish "national priorities" used to select sponsors for the pilot of “tumor board-style” drug reviews that would start before Phase III is completed.

US FDA’s CoGenT Pilot At Risk? Project Orbis Success May Not Save Cell/Gene Therapy Initiative

 
• By Bridget Silverman

Despite following the path of the popular Project Orbis, the cell and gene therapy international collaborative review pilot is being reconsidered by the FDA's new management.

Alzheimer’s Drug Leqembi May Be On Course To India, Kisunla In The Wings

 
• By Anju Ghangurde

Key expert panel go-ahead with a trial waiver put’s Eisai's Alzheimer's therapy on track for a debut in India where tailored pricing will be pivotal. Lilly’s Kisunla is also under regulatory review.

Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 
• By Bridget Silverman

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

More from Pathways & Standards

Post-Approval, Real-Time Monitoring Will Help Approval Standards, US FDA’s Makary Says

 
• By Sue Sutter

The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.

Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 
• By Bridget Silverman

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

US FDA Could Miss User Fee Spending Triggers With House FY 2026 Funding

 
• By Sarah Karlin-Smith and Derrick Gingery

FDA employees are concerned that while the budget authority total proposed for fiscal year 2026 meets the user fee appropriations' triggers, the FDA could have trouble meeting allocation triggers without cuts to non-review work.