blueBird bio Says FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for CALD

By Stephen Nakrosis

Bluebird bio Inc. said Thursday a U.S. Food and Drug Administration committee endorsed elivaldogene autotemcel gene therapy to treat certain patients with early active cerebral adrenoleukodystrophy.

According to bluebird, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee unanimously voted yes on the question "do the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy?"

Bluebird said early active cerebral adrenoleukodystrophy, or CALD, is a "rare, progressive, neurodegenerative disease that primarily affects young boys and causes behavioral, cognitive, and neurological deficits." The company also said eli-cel, if it is approved, will be "the first approved gene therapy to address the underlying genetic cause of disease for patients living with CALD in the U.S."

Write to Stephen Nakrosis at stephen.nakrosis@wsj.com

(END) Dow Jones Newswires

June 09, 2022 18:57 ET (22:57 GMT)

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