Bluebird bio announced the FDA’s acceptance of BLA for beti-cel for patients with β-thalassemia

Bluebird bio, Inc. (BLUE) reported the acceptance of the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review, from the U.S. Food and Drug Administration (FDA), on November 22, 2021. The BLA for beti-cel is based on data from bluebird bio’s Phase 3 studies, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/2 HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia offering an alternative to regular RBC transfusions and iron chelation therapy. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of May 20, 2022. More info: bluebird bio, press release.