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

The FDA’s Advisory Committee has unanimously backed Bluebird bio Inc’s (NASDAQ: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 down 5.89% at $6.55 during the premarket session on the last check Monday after closing 74.4% higher at $6.96 on Friday.