bluebird bio gets FDA priority review for gene therapy

Nov. 22, 2021

bluebird bio announced that the U.S. FDA has accepted the application for priority review of the company's gene therapy designed to treat genetic blood disorders.

The potentially transformative therapy, betibeglogene autotemcel (beti-cel), is designed to treat Beta thalassemia — a blood disorder that reduces the production of hemoglobin. Current standard of care relies on regular red blood cell transfusions and iron management that carry the risk of progressive multi-organ damage and increased risk of death.

According to bluebird, beti-cel works uniquely to help patients produce adult hemoglobin at normal or near-normal levels, which can eliminate their need for chronic transfusions.

If approved, it will be the first one-time treatment option to address the underlying genetic cause of disease and bluebird's first FDA-approved gene therapy.

The BLA for beti-cel is based on data from bluebird bio’s phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), the phase 1/2 HGB-204 (Northstar) and HGB-205 studies, and the long-term follow-up study LTF-303.

It has not been an easy road for the Massachusetts-based biotech, who earlier in the year had to halt trials of its LentiGlobin gene therapy for sickle cell disease after a serious adverse event. The company suspected the issue was linked to the BB305 lentiviral vector used in the LentiGlobin therapy and because the same viral vector was used in its β-thalassemia treatments, several trials were placed on clinical hold.

After further analysis, bluebird determined the adverse event was actually not a case of myelodysplastic syndrome, and the hold was lifted.

The FDA has set a PDUFA goal date of May 20, 2022 for beti-cel.