CBER director does not want to hold back potentially beneficial products, but also warned that patients will have difficult treatment decisions to make since an immune response might prevent a patient from receiving another gene therapy using the same vector ever again.

With the only FDA-approved treatment off the market, the FDA is looking for ideas for development of new preterm birth treatments.

The program bringing Operation Warp Speed-like treatment to rare disease product development could expand if successful, but will require more employees and thus funding commitments from Congress and industry, CBER Director Peter Marks said.

When Janet Woodcock retires early next year, Namandjé Bumpus will take over as No. 2 in command at the FDA. Since joining the agency in 2022 from Johns Hopkins, her profolio has included health disparities, cosmetics, and drug review disputes.

Pink Sheet reporter and editors discuss the FDA acknowledging reports of malignancies in patients who received CAR-T cell immunotherapies, the agency’s policy for using background music in direct-to-consumer advertising, and the FDA’s new incoming principal deputy commissioner.

A recent externally-led PFDD meeting on kidney xenotransplantation, rather than a specific disease, allowed for more tailored patient comments on the procedure, which could impact development plans going forward.