Although clotting factor activity levels may someday be validated as a surrogate endpoint, the currently recommended primary efficacy measure for traditional approval remains annualized bleeding rate, agency says in final guidance.
The US Food and Drug Administration believes factor activity levels may someday be validated as a surrogate endpoint in hemophilia, but for now the agency is sticking with its recommendation of annualized bleeding rate as the basis for traditional approval of gene therapies for the disease.
The agency’s final guidance on hemophilia gene therapy products retains the recommendation in a 2018 draft document that factor...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
