1. Pink Sheet
  2. >>Advanced Technologies
  3. >>Cell & Gene Therapies

Regenerative Medicine Designation More Useful 'Tool' Than CBER’s Marks Initially Anticipated

 
• By Sue Sutter

Biologics center director was skeptical that RMAT would offer much beyond US FDA's existing breakthrough therapy pathway, but he has since come to appreciate the flexibility offered by less stringent designation criteria and leeway in the types of potential confirmatory evidence.

Tool belt
RMAT is a nice tool to have in the regulatory tool belt, CBER's Peter Marks said. • Source: Alamy

The US Food and Drug Administration’s Regenerative Medicine Advanced Therapy (RMAT) expedited pathway has evolved into a more useful “tool” than Center for Biologics Evaluation and Research Director Peter Marks originally expected when the program was established in December 2016 through the 21st Century Cures Act.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Cell & Gene Therapies

US FDA’s Makary Floats Vague ‘Plausible Mechanism’ Approval Pathway For Rare Diseases

 
• By Sarah Karlin-Smith

Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.

EU Pharmacopoeia Adopts ‘Groundbreaking’ Cell Therapy Chapter, Seeks Feedback On Alternative Microbiological Tests

 
• By Neena Brizmohun

A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.

India Signals ‘Systemic Changes’ In CGT Regulatory Approach

 
• By Anju Ghangurde

India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.

Japan Looks To Build Domestic Capacity In New Modalities

 
• By Lisa Takagi

Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.

More from Advanced Technologies

New US FDA Roadmap Moves Closer To Animal Testing Alternatives

 
• By Bridget Silverman

New plan to encourage alternatives to animal testing seeks interagency cooperation to meet more aggressive targets for transformational change than the US FDA’s long-standing program.

Japan Looks To Build Domestic Capacity In New Modalities

 
• By Lisa Takagi

Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.

Pipeline To Product – What Is Driving Cell And Gene Therapy Progress In India?

 
• By Vibha Ravi

US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth.