September advisory committee was less about getting advice and more about calling public attention to AAV gene therapy safety issues, OTAT director Wilson Bryan says. FDA is not ready to set a cap on vector dose but could consider doing so down the road.
The US Food and Drug Administration’s recent advisory committee meeting on adeno-associated virus (AAV) vector-based gene therapies succeeded in bringing transparency to safety issues that have troubled the field and resulted in the scientific community taking a “hard look” at such toxicities, Office of Tissues and Advanced Therapies director Wilson Bryan said.
The two-day meeting in September of the Cellular, Tissue and Gene Therapies Advisory Committee was less about getting advice and...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
