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Bluebird’s Brain Disease Therapy Wins FDA Advisers’ Support

  • FDA staff had expressed concerns about data interpretation
  • Patients’ families plead for recommendation in meeting
Bluebird Bio headquarters in Cambridge, Mass.

Bluebird Bio headquarters in Cambridge, Mass.

Photographer: Kristoffer Tripplaar/Sipa USA/AP
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Bluebird Bio Inc.’s gene therapy for a brain-wasting disease received support from a panel of outside advisers to US regulators, giving the treatment’s prospects for clearance a key boost. 

A Food and Drug Administration advisory committee voted 15-0 in support of Bluebird’s eli-cel therapy for cerebral adrenoleukodystrophy, or CALD, a rare genetic condition that afflicts young boys, causing rapid deterioration and death. Panelists said the potential benefits of the therapy outweigh risks that may include cancer. The vote isn’t binding, but the FDA usually follows recommendations from the panel, the Cellular, Tissue, and Gene Therapies Advisory Committee