The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for patients with β-thalassemia who require regular red blood cell transfusions.

Beti-cel is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. The treatment is expected to correct the deficiency of adult hemoglobin observed in patients with β-thalassemia.

The application is supported by data from the phase 3 Northstar-2 ( Identifier: NCT02906202) and Northstar-3 ( Identifier: NCT03207009) studies, along with two phase 1/2 studies ( Identifier: NCT01745120, NCT02151526) and a long-term follow-up study ( Identifier: NCT02633943).

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The ongoing multicenter, single-arm, single-dose Northstar-2 (n=23) and Northstar-3 (n=18) studies evaluated the efficacy and safety of beti-cel administered by intravenous infusion in adult, adolescent, and pediatric patients with β-thalassemia following myeloablative conditioning with busulfan. 

Among 36 evaluable patients, findings showed that 89% (n=32) achieved transfusion independence across all ages and genotypes, including patients as young as 4 years of age and those with the most severe genotypes (Northstar-2: median follow-up 24.3 months; Northstar-3: median follow-up 23 months). Transfusion independence was defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average hemoglobin of at least 9g/dL.

As for safety, infusion-related reactions (abdominal pain, hot flush, dyspnea, tachycardia, and noncardiac pain) and cytopenias (thrombocytopenia, leukopenia, and neutropenia) were observed in patients treated with beti-cel.

“The FDA’s acceptance of our BLA for beti-cel brings us one step closer to potentially providing a one-time treatment that can address the underlying cause of β-thalassemia and offer patients freedom from regular transfusions,” said Andrew Obenshain, CEO, bluebird bio.

A Prescription Drug User Fee Act (PDUFA) target date of May 20, 2022 has been set for the application.

The FDA previously granted Orphan Drug and Breakthrough Therapy designations to beti-cel for this indication.


  1. Bluebird bio announces FDA Priority Review of Biologics License Application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions. News release. Bluebird bio, Inc. Accessed November 22, 2021.
  2. Betibeglogene autotemcel (beti-cel) one-time gene therapy for β-thalassemia continues to demonstrate durable efficacy across pediatric and adult patient populations and all genotypes in data presented at EHA2021 Virtual. News release. Bluebird bio, Inc. June 11, 2021. Accessed November 22, 2021.

This article originally appeared on MPR