The new designation could streamline development of multiple products that use the same backbone, said Nicole Verdun, director of the US FDA’s Office of Therapeutic Products.
Office of Therapeutic Products Director Nicole Verdun sees the new platform technology designation as another pathway to expedited approval of gene therapies from the same sponsor and eventually, multiple sponsors.
The 2022 Food and Drug Omnibus Reform Act, which renewed several US Food and Drug Administration user fee programs, created...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
