The Food and Drug Administration (FDA)’s Cellular, Tissue, and Gene Therapies Advisory Committee voted in favor of the approval of betibeglogene autotemcel (beti-cel) for patients with beta-thalassemia who require regular red blood cell transfusions.

Beti-cel is an investigational 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 FDA panel’s recommendation was based on 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 single-arm, single-dose Northstar-2 and Northstar-3 studies evaluated the efficacy and safety of beti-cel administered by intravenous infusion in adult, adolescent, and pediatric patients with beta-thalassemia following myeloablative conditioning with busulfan.

As of the data cutoff date of August 2021, 89% (n=34/38) of evaluable patients achieved transfusion independence across all ages and genotypes, 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. Infusion-related reactions (abdominal pain, hot flush, dyspnea, tachycardia, and noncardiac pain) and cytopenias (thrombocytopenia, leukopenia, and neutropenia) were reported in patients treated with beti-cel.

When asked if the benefits of beti-cel outweigh the risks for the treatment of patients with transfusion-dependent beta-thalassemia, the committee voted 13 “yes” and 0 “no”.

Although not bound by the panel’s recommendations, the FDA does take them into consideration when making decisions on approval. The Biologics License Application (BLA) for beti-cel is currently under Priority Review by the FDA with a Prescription Drug User Fee Act (PDUFA) target date of August 19, 2022. 

“Today’s advisory committee recommendation is recognition of the substantial body of clinical data that support beti-cel as a potentially curative treatment option for these patients. We are grateful to the members of the beta-thalassemia community who contributed to today’s discussion and remain committed to working with the FDA as it completes its review of the beti-cel Biologics License Application,” said Andrew Obenshain, CEO, bluebird bio. 

The Company’s other lentiviral vector gene therapy, elivaldogene autotemcel (eli-cel), was also recently endorsed for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor.


FDA advisory committee unanimously supports beti-cel gene therapy for people with beta-thalassemia who require regular red blood cell transfusions. News release. bluebird bio, Inc. Accessed June 10, 2022.

This article originally appeared on MPR