US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final product’ in making the determination.
The US Food and Drug Administration’s draft guidance on determining orphan exclusivity for gene therapy products doesn’t appear to include enough details to offer complete reassurance to sponsors worried about a getting a designation, but the overall tone suggests that the agency is going to try to provide as many designations as possible.
The draft guidance, released on 28 January along with a spate of final guidances on other gene therapy topics,...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
