FDA advisers unanimously endorse Bluebird’s gene therapy for rare disorder
Advisers to the Food and Drug Administration voted unanimously in favor of approving an investigational gene therapy from Bluebird Bio on Thursday, concluding that its benefits for children with a rare and deadly disorder outweigh a demonstrable risk of causing cancer.
The FDA’s independent advisers voted 15-0 to recommend Bluebird’s eli-cel, a one-time treatment for cerebral adrenoleukodystrophy, a genetic neurological disorder that affects young boys. The agency, which has promised to make a final decision on eli-cel by Sept. 16, is not required to follow the advice of its advisory committees.
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