US FDA's Peter Marks says the field is robust, but hedges on approval predictions for the coming years because of pandemic’s effect.
Gene therapy development and approval likely was slowed by the coronavirus pandemic, which could endanger projections for the explosion in the sector going forward.
In calendar year 2019, the US Food and Drug Administration received 243 IND applications for gene therapies, which was followed...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
