FDA accepts beti-cel BLA for priority review in β-thalassemia

The US Food and Drug Administration has accepted the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review.

Developed by US biotech bluebird bio (Nasdaq: BLUE), beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions. bluebird’s shares initially leapt 12%, but closed down 1.5% at $10.54 yesterday.

If approved, beti-cel will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia in the USA - 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.