bluebird bio (BLUE) Stock Price: Why It Surged Over 70% Today

By Amit Chowdhry ● Jun 13, 2022
  • The stock price of bluebird bio Inc (NASDAQ: BLUE) increased by over 70% pre-market today. This is why.

The stock price of bluebird bio Inc (NASDAQ: BLUE) increased by over 70% pre-market today. Investors have been responding positively to bluebird bio recently announcing the outcome of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) discussion of betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions.

On the question “Do the benefits of beti-cel outweigh the risks for the treatment of subjects with transfusion-dependent beta-thalassemia?” the CTGTAC had voted 13 (yes) to 0 (no).

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. And 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 the 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. And 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. And 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. 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 unanimously endorsed eli-cel, an investigational LVV gene therapy for the treatment of cerebral adrenoleukodystrophy, in a 15 to 0 vote.

In addition to granting the beti-cel BLA priority review, the FDA had previously granted beti-cel Orphan Drug status and Breakthrough Therapy designation. bluebird bio is eligible to receive a priority review voucher upon potential approval of beti-cel.


“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. 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.”

— Andrew Obenshain, chief executive officer, bluebird bio

“We are pleased that the advisory committee recognized the significant potential of LVV gene therapies to address severe unmet medical needs – demonstrated over more than a decade of research and more than 500 patient-years of experience – as well as the urgent need for improved treatments. We thank the FDA for convening this discussion on bluebird bio’s LVV gene therapies and look forward to the completion of the Agency’s review.”

— Anne-Virginie Eggimann, chief regulatory officer, bluebird bio

Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.