CBER’s director says manufacturing and regulatory issues need to be resolved to ensure the gene therapy sector thrives.
Peter Marks believes the gene therapy sector needs all the help available to boost product development and lower costs, including from the US Food and Drug Administration.
The director of the FDA’s Center for Biologics Evaluation and Research Director said 6 November “I think I would pull...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
