Bluebird submits BLA to FDA for β-thalassaemia gene therapy

Bluebird bio announced it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassaemia who require regular red blood cell transfusions, across all β genotypes. Beti-cel is already cleared for market in Europe, where it's sold under the brand name Zynteglo. The BLA submission for beti-cel is based on data from Phase 1/2 and Phase 3 Northstar studies, which represent more than 220 patient-years of experience with beti-cel. The FDA previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of transfusion-dependent β-thalassemia. If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the United States. Source: bluebirdbio press release