FDA Advisory Committee Backs Bluebird Bio's Blood Disorder Gene Therapy

  • The FDA's Advisory Committee has unanimously backed Bluebird bio Inc's BLUE gene therapy for a rare blood disorder.
  • The panel of 13 voted unanimously for betibeglogene autotemcel (beti-cel) as a one-time gene therapy to treat Beta-thalassemia patients dependent on blood transfusions.
  • If approved, beti-cel will be the first potentially curative gene therapy option for beta-thalassemia who require regular red blood cell transfusions and the first ex-vivo LVV gene therapy available in the U.S.
  • The decision comes less than 24 hours after the same committee, in a surprise move, endorsed approval of bluebird's other gene therapy,elivaldogene autotemcel (eli-cel), for a rare neurological disease despite concerns that the treatment may cause cancer.
  • Related: FDA Sounds Positive On Bluebird's One Gene Therapy But Doubts Another.
  • On June 9, the CTGTAC unanimously endorsed eli-cel for cerebral adrenoleukodystrophy in a 15 to 0 vote.
  • The FDA is expected to decide on beti-cel's fate by August 19 and eli-cel's by September 16.
  • Price Action: BLUE shares are up 58.45% at $5.89 during the premarket session on the last check Monday.

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