Two outside groups aim to help CBER on gene therapy questions from ‘N of one’ to markers for hemophilia.
After more than two years in the making, two outside gene therapy consortia are underway in the US to help broaden the consensus starting point on discussions with CBER on characterization assays and the appropriate use of surrogate endpoints.
The recent formal introductions of two AAV gene therapy consortia permit FDA’s Center for Biologics Evaluation and Research to consolidate...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
