BlueBird Bio Shares Soar 53.30% After FDA’s Committee Approves its Beti-cel Gene Therapy

Bluebird Bio Stock surges nearly 53% after an advisory committee to the U.S. Food and Drug Administration votes to approve beti-cel gene therapy for people with beta-thalassemia.

Source: Bluebirdbio.

BLUE up +53.30%, IONS down -2.53%

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 13-0 in favor of the company’s beti-cel for treating people with beta-thalassemia who require red blood cells (RBC) transfusions.

Patients diagnosed with the most severe form suffer from severe anemia and lifelong dependence on red blood cell transfusions that patients experience each 2-5 weeks.

The advisory committee’s recommendation was dependent on the Biologics License Application (BLA) under priority review by the FDA, with a decision target date set for August 19, 2022.

The FDA initially granted beti-cel Orphan Drug status and Breakthrough Therapy designation and bluebird bio are eligible to obtain a priority review voucher based on approval of beti-cel.