Development guidance from CBER recommends comparing investigational gene therapy products to existing prophylaxis treatment.
CBER wants proposed gene therapies for hemophilia to show they are no worse than existing therapies, potentially changing the paradigm with single-dose treatments in development.
In its first ever draft guidance on hemophilia therapy development, CBER said that gene therapies could potentially mark a...
The official separation of those impacted by the reduction-in-force and some others who are leaving the agency are not yet reflected in CDER employee gains and losses data.
Development of the user fee commitment letter largely takes place behind closed doors between industry and FDA. Outside groups see the upcoming negotiation cycle as a chance to change this and other agency information blocks by reminding Commissioner Makary of his commitment to transparency.
Senate appropriators also put FDA on notice regarding recent meetings and compliance with the Federal Advisory Committees Act, signal positive news on rare pediatric diseases, and offer hints of other agency areas they have close eyes on.
European regulators must work hard to maintain and build vital but fragile trust in science and processes, said speakers at an event celebrating the European Medicines Agency 30th anniversary.
