Comment by arjie
1 day ago
It’s in the OP:
> Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program
That appears to be a late 2025 program based on the dates on the FDA page which would make it a Trump administration policy change. I don’t have any real knowledge here though. I’m just a guy clicking links. But if this is a policy change that’s exciting for approval of the other gene therapies for related conditions once the technology is solved.
0: https://www.fda.gov/industry/commissioners-national-priority...
I do agree that FDA was quite risk averse before especially in areas where the risk - reward would be better for terminally ill or other patients. Having said that this is a different kind of risk reward treatment where the patient might themselves like to try it even if the risks were elevated compared to other post Phase 3 solutions.
AFAIK critics pointed this to be a form of regulatory capture as well.