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Just one day after it received a positive recommendation for one gene therapy, bluebird got another. This one is for betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions.

The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 13 (yes) to 0 (no) on the question “Do the benefits of beti-cel outweigh the risks for the treatment of subjects with transfusion-dependent beta-thalassemia?”

This is great news for people with the disease, as well as for bluebird, which in April announced it would eliminate about 30% of its staff as part of a restructuring plan designed to generate $160 million in savings over two years.  A month earlier the company had acknowledged in its Form 10-K: “There is substantial doubt regarding our ability to continue as a going concern.”  While good news had helped the company reach a $12 billion market valuation in 2018, a series of woes caused the setback. This recent progress could turn things around.

“Despite advances in care, people living with the most severe form of beta-thalassemia still require frequent transfusions of healthy red blood cells to survive, tethering them to the healthcare system for life and increasing their risk for severe complications and early death,” said Andrew Obenshain, chief executive officer, bluebird bio.

He added that, “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.”

Beta-thalassemia is a rare genetic blood disease caused by mutations in the beta-globin gene and is characterized by significantly reduced or absent adult hemoglobin production. Patients with the most severe form experience severe anemia and lifelong dependence on red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks.

Despite advances in treatment and improved transfusion techniques, transfusions only temporarily address symptoms of anemia and people with beta-thalassemia who require regular transfusions have an increased risk for morbidity and mortality due to treatment- and disease-related iron overload and its complications. Data from the Cooley’s Anemia Foundation indicate that the median age of death of U.S. transfusion-dependent beta-thalassemia patients who died during the last decade was just 37 years.

The advisory committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a decision goal date set for August 19, 2022. The BLA 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 as of March 2021. Additionally, as of the latest data cutoff date (August 2021), data from bluebird bio’s clinical development program represent 240 patient-years of experience with beti-cel and the longest available follow-up data in beta-thalassemia patients requiring regular RBC transfusions treated with a one-time gene therapy.

On June 9, the CTGTAC endorsed eli-cel, an investigational lentiviral vector-based gene therapy for the treatment of cerebral adrenoleukodystrophy, in a 15 to 0 vote.

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