BLUE - Bluebird bio gets FDA AdCom backing for rare blood disorder therapy

An independent panel of experts of the FDA unanimously recommended a gene therapy developed by Bluebird bio (NASDAQ:BLUE) for regulatory authorization as a treatment for ?-thalassemia on Friday. FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 13-0 in favor of beti-cel as a potential therapy for the rare blood disorder. The FDA's advisory committees issue non-binding recommendations. However, the regulator usually follows them before making a final decision on authorizations. The FDA is expected to make its decision on beti-cel on or before y Aug. 19. Just a day earlier, the panelists endorsed eli-cel, another gene therapy candidate developed by the company for children with rare neurodegenerative disease cerebral adrenoleukodystrophy (CALD) for which a final decision is expected by Sept. 16.

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Bluebird bio gets FDA AdCom backing for rare blood disorder therapy