BLUE - bluebird bio jumps 12% on FDA acceptance of beti-cel application for priority review

bluebird bio (NASDAQ:BLUE) gains 12% premarket after the FDA accepted for priority review, the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel). The agency has set a PDUFA goal date of May 20, 2022. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with ?-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 in U.S.—offering an alternative to regular RBC transfusions and iron chelation therapy. The BLA is based on data from 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. The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation.

For further details see:

bluebird bio jumps 12% on FDA acceptance of beti-cel application for priority review