A panel of advisers to the Food and Drug Administration voted unanimously to recommend approval of an investigational gene therapy from Bluebird Bio on Friday, a one-time treatment that could be a cure for some patients with a rare blood disorder.
The group of independent experts voted 13-0 that the benefits of Bluebird’s gene therapy outweigh its risks for patients with beta-thalassemia, a disease that leads to severe anemia and requires patients to undergo frequent blood transfusions. The FDA is not required to follow the recommendations of its advisers, though it typically does. The agency has promised to render a final decision on Bluebird’s gene therapy, called beti-cel, by Aug. 19.
“The efficacy of this therapy is outstanding,” said Jeannette Lee, a panelst and professor of biostatistics at the University of Arkansas for Medical Sciences. ”The opportunity to be transplant-independent is really life-changing for patients.”
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