US FDA’s Gottlieb tells J.P. Morgan that curative gene therapies may be ripe for accelerated approval while those targeting symptoms may require more conventional development programs; forthcoming agency guidance will articulate this divide specific to neurodegenerative diseases, he says.
The US FDA will soon clarify the regulatory pathways and evidentiary requirements for gene therapies for neurodegenerative diseases based upon a treatment’s intended effect, Commissioner Scott Gottlieb said Jan. 8.
In a keynote address to the J.P. Morgan conference in San Francisco, Gottlieb said the agency intends to distinguish when...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
