Some worries about heritable genetic modifications are subsiding and Center for Biologics Evaluation and Research Director Peter Marks said sponsors should consider the US for regulatory advice and clinical trials in the space.
The US Food and Drug Administration’s growing comfort with gene editing has sparked a more relaxed regulatory approach to the products.
Gene editing is a concerning subject for the FDA because the products could unknowingly cause heritable genetic modifications that harm...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
