BLUE - Bluebird bio neuro disorder gene therapy gets FDA panel nod awaits meeting on another

Bluebird bio (NASDAQ:BLUE) said a panel of the U.S. Food and Drug Administration (FDA) voted in favor of its gene therapy candidate elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). The company's stock had been halted for trading by Nasdaq on June 9 for 2 days (including today) ahead of the FDA committee meeting. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 15-0 in favor that the benefits of eli-cel outweighed the risks, for treating  any sub-population of children with early active CALD, the company said in a June 9 press release. CALD is a neurodegenerative disease that mainly affects young children and leads to irreversible loss of neurologic function and death, said the company. The panel's endorsement is win for Bluebird as the eli-cel clinical program was placed on a clinical hold following a Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS) in August 2021. Following this, two additional cases of

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Bluebird bio neuro disorder gene therapy gets FDA panel nod, awaits meeting on another