Fierce Biotech May 8, 2024
Accelerated approval—controversial, yes—but absolutely necessary for small, pre-revenue biotechs trying to find new treatments for small patient populations.
That’s according to the FDA’s Peter Marks, M.D., Ph.D., who was speaking at the American Society of Gene and Cell Therapy annual meeting Wednesday.
Marks, director of the Center for Biologics Evaluation and Research (CBER), provided his thoughts on how to reduce costs for the many small companies working in gene therapy; an area with great potential, but also risk.
Using a biomarker to obtain accelerated approval is key to get an initial approval for therapies that address devastating diseases, rather than wait for a clinical endpoint to show efficacy in a longer trial.
“The wherewithal to do a three-year study...
Today's Sponsors
Today's Sponsor