Bluebird bio's gene therapy for blood disorder wins FDA panel backing
FDA panel backs Bluebird gene therapy for blood disorder hours after endorsing another Bluebird therapy for a rare neurological disease despite safety risks.
Advisors to the US Food and Drug Administration backed on Friday the Bluebird bio's (BLUE.O) treatment for a rare blood disorder, in a vote of confidence in gene therapies and bolstering the company beset by regulatory setbacks.
The backing comes less than 24 hours after the same committee endorsed approval of bluebird's gene therapy, eli-cel, for a rare neurological disease even though there have been some concerns that the therapy may lead to cancer.
The panel of 13 outside experts voted unanimously on Friday for beti-cel, as a one-time gene therapy that treats Beta-thalassemia patients who rely on blood transfusions.
The FDA is to decide on beti-cel's fate by August 19 and eli-cel's by September 16. The agency is not required to abide by its advisors' recommendations, although it normally does.
Both products are expected to be launched this year if the FDA approves the therapies.
"Our expectation is that the product should be treating first patients by early fourth quarter," Chief Executive Officer Andrew Obenshain told Reuters.
The therapy's efficacy data was "outstanding", as per Panel member Jeannette Yen Lee, who added that "the opportunity to be transplant-independent is really life-changing for the patient."
Sarah Baqueri-Connolly, a parent of a Beta-thalassemia patient who died in 2015, said, "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."
“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.