bluebird bio progresses BLA for betibeglogene autotemcel gene therapy

US biotech bluebird bio (Nasdaq: BLUE) says it has completed the rolling submission of its Biologics License Application (BLA) to the US Food and Drug Administration for betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell (RBC) 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 (TDT). If approved, beti-cel will be the first hematopoietic (blood) stem cell (HSC) ex-vivo gene therapy for patients in the USA.

In Europe, the cell therapy is marketed under the brand name Zynteglo. However, due to a poor uptake of gene therapies as national health technology regulators won’t agree acceptable prices, bluebird plans an orderly wind down of its operations in Europe and to explore how to give patients in Europe access to its gene therapies, including potentially out-licensing the ex-US rights to its three lead products to a company with European experience and capabilities.