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Supporting Vote

An advisory committee to the US Food and Drug Administration has voted to support Bluebird Bio's gene therapy for cerebral adrenoleukodystrophy, according to Bloomberg.

Cerebral adrenoleukodystrophy (CALD) is a rare, X-linked genetic condition affecting mostly boys that leads to progressive and irreversible neurodegenerative disease. About half of individuals with CALD die within five years of the onset of symptoms. Bluebird Bio's eli-cel (elivaldogene autotemcel) introduces functional copies of the ABCD1 gene using a Lenti-D lentiviral vector.

Last year, the FDA placed the company's eli-cel trial on a clinical hold after patients who received treatment later developed myelodysplastic syndrome. Bloomberg notes that the trial is still on hold, "creating an unusual situation for a therapy that's under consideration for approval."

Still, the FDA advisory panel voted 15-0 in support of the treatment. According to Bloomberg, some panelists suggested offering the treatment to patients without other options and all underscored the need for close monitoring of patients receiving the treatment.