Final gene therapy IND CMC guidance clarifies expectations on process evolution, distinguishing drug substances from products, cGMP oversight and more.
The US Food and Drug Administration repeatedly emphasized in the final version of its guidance on chemistry, manufacturing and control information for investigational gene therapies its expectation that sponsors will continually improve manufacturing processes and analytics during development – and that they will update their investigational new drug applications (INDs) accordingly.
The agency sprinkled the final guidance document with observations and suggestions that were not in the July 2018
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
