Two required confirmatory studies will bolster application that relied on historical controls for orphan neurodegenerative disease.
The US FDA-approved labeling of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) stayed consistent with the agency’s position at a June advisory committee meeting, but with one notable twist: the gene therapy received accelerated approval, an option that was not mentioned in the committee briefing documents or meeting transcript.
Neuroscience is not a field that has had much use for accelerated approval, with Biogen, Inc.’s Aduhelm standing as...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
