FDA priority review of BLA for beti-cel gene therapy for patients with beta-thalassemia.

Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with beta-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 beta-thalassemia in the U.S.—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.

The BLA for beti-cel is based on data from bluebird bio’s Phase III studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the Phase 1/II HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303. Together, these studies represent more than 220 patient-years of experience with beti-cel. As of March 9, 2021, the results include a total of 63 pediatric, adolescent and adult patients, including long-term efficacy and safety results in two patients with more than seven years follow-up. Additional data through August 2021 will be presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 11-14, 2021.