Gene Therapy Reviews Return To US FDA Advisory Committee Schedule
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.
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Although there were no cases of hematological malignancy reported and no evidence of insertional oncogenesis in patients treated with beti-cel, FDA raises a theoretical risk due to delayed platelet engraftment, as well as malignancies seen in other lentiviral vector programs.
Interpretation of efficacy from single-arm studies in patients with cerebral adrenoleukodsytrophy complicated by comparability with external controls and limited follow-up, FDA says in advisory committee briefing documents which also cite three cases of myelodysplastic syndrome and raise broader questions about lentiviral vector safety.
Office of Tissues and Advanced Therapies seeks to optimize its organizational structure and adopt more group-level communications, instead of one-on-one interactions with sponsors, to keep up with the massive growth in cell and gene therapies, director Wilson Bryan says.