Pink Sheet infographic looks at the nascent space of gene therapy accelerated approvals, where confirmatory trial timelines can run many years beyond approval due to disease rarity and a focus on durability of response.
The US Food and Drug Administration has granted accelerated approval to four gene therapies to date, including three anti-cancer CAR-T products and bluebird bio’s cerebral adrenoleukodystrophy treatment Skysona (elivaldogene autotemcel).
New confirmatory trials were required for all of them. In the case of Skysona, the last study report is not...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
