US FDA Commissioner teases endpoints, suggests accelerated approval plan and post-marketing considerations for gene therapies targeting hemophilia.
US FDA Commissioner Scott Gottlieb outlined an accelerated approval path for gene therapies in hemophilia, offering a potential preview of agency expectations for the forthcoming applications.
In a May 22 speech at the Alliance for Regenerative Medicine's annual board meeting, Gottlieb began describing the contents of...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
