bluebird bio completes rolling submission to FDA for beti-cel in Beta-thalassemia

The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the United States.

The BLA submission for beti-cel is based on data from patients treated in bluebird bio studies, including the Phase III HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies, and the Phase I/II HGB-204 (Northstar) and HGB-205 studies. 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 who have been treated with beti-cel across beta0/beta0 and non-beta0/beta0 genotypes. The data include two patients with up to seven years of follow-up, eight with at least six years of follow-up and 19 with at least five years of follow-up, and were most recently shared during the 26th Annual Congress of the European Hematology Association (EHA2021 Virtual).