FDA committee votes in favor of Bluebird’s gene therapy for rare neurodegenerative disease

A Food and Drug Administration advisory committee on Thursday voted 15-0 that the benefits of Bluebird Bio Inc.’s experimental gene therapy outweigh the risks for patients younger than 18 years old who are being treated for early cerebral adrenoleukodystrophy. Bluebird’s stock was halted on Thursday while the committee discussed and then voted on elivaldogene autotemcel. In a news release, the company said that if the therapy receives FDA approval, it “will be the first and only gene therapy for the treatment of early active CALD.” Bluebird’s stock is also set to be halted on Friday while the committee discusses the risks and benefits of another gene therapy being developed by Bluebird. The company has asked the FDA to approve betibeglogene autotemcel as a treatment for beta thalassemia. Bluebird’s stock is down 61.9% this year, while the broader S&P 500 has declined 12.7%.

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