Bluebird bio’s gene therapy for blood disorder gets FDA panel backing

Reuters - 10/06/2022 - Bluebird bio's treatment for a rare blood disorder received backing from advisers to the U.S. Food and Drug Administration on Friday, in a vote of confidence in gene therapies and bolstering the company beset by regulatory setbacks.

The decision comes less than 24 hours after the same committee in a surprise move endorsed approval of bluebird's other gene therapy, eli-cel, for cerebral adrenoleukodystrophy, despite concerns that the treatment may cause cancer.

The panel of 13 outside experts on Friday voted unanimously for beti-cel as a one-time gene therapy to treat Beta-thalassemia patients dependent on blood transfusions.

A favorable decision was widely anticipated after FDA reviewers said beti-cel demonstrates "clinically meaningful" benefit in patients in briefing documents published on Tuesday.

Panel member Jeannette Yen Lee said the therapy data was "outstanding", adding that "the opportunity to be transplant independent is really life changing for the patient."

"We've personally watched friends and family participate in gene therapy trials and be cured of this painful disease. They no longer need blood transfusions. They're finally able to live a life that doesn't revolve around hospital stays, blood draws medications," said Sarah Baqueri-Connolly, parent of a Beta-thalassemia patient who passed away in 2015.

"I believe the therapy gives patients and their families hope, a hope that we didn't have."

The back-to-back positive decisions are a big boost for bluebird, which had in March flagged "going concern" doubts following regulatory setbacks.

The company's shares had fallen 72% this year through Monday's close, before rising about 20% on Tuesday after FDA reviewers said data supports the effectiveness of beti-cel. Trading in bluebird bio's stock was halted for Thursday and Friday.

The FDA is expected to decide on beti-cel's fate by Aug. 19. The agency is not required to follow its advisers' recommendation, although it usually does.

By Reuters Staff



Posted on 10 June 202221 June 2022