Expedited approval pathway had been a secondary option when traditional approval was not possible, but the CBER director wants surrogate endpoints increasingly considered from the beginning, especially with pediatric rare disease gene therapies.
Gene therapy sponsors now should expect accelerated approval to be on the table from the beginning of the development process, rather than as an alternative to a traditional pathway.
Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, said the change in...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
