Regenerative Medicine Designation More Useful 'Tool' Than CBER’s Marks Initially Anticipated
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.
You may also be interested in...
Over the first five years of the regenerative medicine advanced therapy designation, analysis by the Pink Sheet finds the 40% success rate of requests is comparable to the broader breakthrough therapy designation, despite less stringent RMAT criteria.
After missing user fee date due to inspection delay, US FDA clears the CAR-T therapy for treatment of adults with relapsed or refractory large B-cell lymphoma.
Regulatory review resource-intensive cell and gene therapy licensing applications are rolling toward US FDA along expedited pathways, while complete response letters and missed goal dates start to stack up under pandemic inspection constraints.