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

[June 11, 2022] 

By Mrinalika Roy

(Reuters) -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 a rare neurological disease 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.

The FDA is expected to decide on beti-cel's fate by Aug. 19 and eli-cel's by Sept. 16. The agency is not required to follow its advisers' recommendation, although it usually does.

If the FDA approves the therapies, bluebird expects to launch both the products by this year.

"Our expectation is that the product should be treating first patients by early fourth quarter," Chief Executive Officer Andrew Obenshain told Reuters.

Panel member Jeannette Yen Lee said the therapy's efficacy 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," said Sarah Baqueri-Connolly, parent of a Beta-thalassemia patient who passed away in 2015.

"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.

“Hopefully, with approval will come some priority review vouchers for these products that will provide some non dilutive funding. And we'll also look at other ways of raising funding," CEO Obenshain said.

(Reporting by Mrinalika Roy in Bengaluru; Editing by Maju Samuel)

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