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FDA Panel Endorses Bluebird Bio's Beti-cel Gene Therapy For Rare Blood Disease

An advisory committee to the U.S. Food and Drug Administration voted unanimously to recommend approval of bluebird bio Inc.'s (BLUE) beti-cel gene therapy for people with beta-thalassemia who require regular red blood cell transfusions.

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted 13-0 in favor of the company's betibeglogene autotemcel or beti-cel for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions.

Beta-thalassemia is a rare genetic blood disease caused by mutations in the beta-globin gene and is characterized by significantly reduced or absent adult hemoglobin production. Patients with the most severe form experience severe anemia and lifelong dependence on red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks.

The advisory committee's recommendation was based on the Biologics License Application (BLA) currently under priority review by the FDA with a decision goal date set for August 19, 2022.

In addition to granting the beti-cel BLA priority review, the FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation. bluebird bio is eligible to receive a priority review voucher upon potential approval of beti-cel.

On Thursday, the FDA panel, which scrutinized the company's investigational gene therapy Elivaldogene autotemcel, or Eli-cel in short, proposed for the treatment of early active cerebral adrenoleukodystrophy in patients less than 18 years of age without a matched sibling donor, unanimously recommended its approval.

Cerebral adrenoleukodystrophy is a rare neurodegenerative disease primarily affecting young children that can lead to progressive, irreversible loss of neurologic function and death.

The FDA decision on Eli-cel is expected on September 6, 2022.

Trading of the company's common stock was halted on Thursday and Friday.

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