FDA comments on biomarkers as surrogate endpoints for AA
March 21, 2023 - ".... for these gene therapies to make it through clinical trials in a “relatively facile manner”, the agency has to make use of what it has at its disposal to grant the therapies accelerated approval. This includes using surrogate endpoints like biomarkers to measure expected clinical benefit.
After accelerated approval is granted, the companies would then be required to confirm the therapy’s safety and efficacy through trials with measured endpoints. "
https://www.biospace.com/article/fda-official-backs-accelerated-approval-for-gene-therapies-ahead-of-sarepta-s-dmd-adcomm/