Although US FDA had received 22 adverse event reports by end of 2023, CAR-T products’ benefit-risk profiles remain ‘incredibly beneficial’ and the FDA has no concerns about approved oncology uses, Center for Biologics Evaluation and Research Director Peter Marks says.
Chimeric antigen receptor T-cell (CAR-T) therapies continue to have the blessing of the US Food and Drug Administration’s top cell and gene therapy regulator despite the agency’s high-profile safety alert about adverse event reports of T-cell malignancies.
The rate of T-cell malignancy reports with the products in the oncology setting is not “overly concerning,” and CAR-T product...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
