RE:FDA's Head of CBER wants faster approvals with SE/biomarkers" Safety data is a red line for Marks, however. He asserted that he would never overrule reviewers if there were questions about safety. Sarepta’s gene therapy, for example, had 13 serious side effects in 85 patients but no deaths or discontinuations due to the treatment. Duchenne advocates who spoke at advisory hearings for the therapy were emphatic that the side effects were dwarfed by the risk of disease progression. "
Marks also said candidly that as a center director at the agency, he’s more willing to take risks than staffers, saying that it’s “natural as a regulator to be somewhat conservative.”
https://www.fiercebiotech.com/biotech/fdas-marks-i-contradict-fda-reviewers-trepidation