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.
“This treatment isn’t a cure, but it at least gives these boys time until hopefully one day we can come up with something better for them,” said Stephanie Keller, a professor of pediatrics at Emory University and a member of the advisory committee.
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