Posted by Michael Wonder on 21 Sep 2021
bluebird bio submits biologics license application to FDA for betibeglogene autotemcel (beti-cel) gene therapy for patients with β-thalassaemia who require regular red blood cell transfusions
21 September 2021 - Biologics license application submission based on data from Phase 1/2 and Phase 3 Northstar studies, which represent more than 220 patient-years of experience with beti-cel.
bluebird bio today announced it has completed the rolling submission of its biologics license application to the US FDA for betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and paediatric patients with β-thalassemia who require regular red blood cell transfusions, across all genotypes.
The FDA previously granted beti-cel orphan drug status and breakthrough therapy designation for the treatment of transfusion-dependent β-thalassemia.
