The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.
The upcoming two-day review in June for two of bluebird bio’s gene therapies will end a surprising fallow period for reviews by the US Food & Drug Administration’s external Cellular, Tissue & Gene Therapies Advisory Committee (CTGTAC).
Betibeglogene autotemcel (beti-cel) for beta-thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy in patients under 18 will go before CTGTAC...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
