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.

Benefits of independence from exogenous insulin outweighed the risks from long-term immunosuppression necessary with the allogeneic pancreatic islet cellular therapy, majority of panelists said, but they urged the FDA to limit the indication to a very small subpopulation of patients.

Proportion of subjects with freedom from exogenous insulin ‘might support the efficacy’ of the allogeneic islet cellular therapy, US FDA says after concluding that issues in two studies limit interpretability of primary analyses based on severe hypoglycemic events and HbA1c levels. An advisory committee will weigh clinical efficacy, safety and product characterization issues.

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker