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bluebird bio Submits BLA for Gene Therapy to Treat β-thalassemia
bluebird bio based its BLA submission for the gene therapy on data from four Phase 3 clinical trials, which found that 89% of patients across all ages achieved transfusion dependence.
bluebird bio recently announced the completion of its rolling submission of its Biologics License Application to FDA for betibeglogene autotemcel (beti-cel) gene therapy in patients with β-thalassemia.
The therapy is intended for pediatric, adolescent, and adult patients with β-thalassemia who require regular red blood cell (RBC) transfusions across all genotypes.
Previously, FDA granted beti-cel Orphan Drug Status and Breakthrough Therapy designation to treat transfusion-dependent β-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.
“With this submission, we are one step closer to bringing a potentially transformative gene therapy to people living with TDT and their families,” Andrew Obenshain, president of severe genetic diseases at bluebird bio, said in the announcement.
“At bluebird bio, we have a deep understanding of gene therapies, built over a decade of research and development in severe genetic diseases. We look forward to working with the FDA on its review of this BLA as we realize the promise that one-time gene therapies hold for patients,” Obenshain continued.
Transfusion-dependent β-thalassemia (TDT) is a severe genetic disease caused by mutations in the β-globin gene that cause reduced adult hemoglobin, resulting in chronic anemia and lifelong dependence on red blood cell transfusions, a bluebird bio spokesperson explained.
Individuals living with this disease generally require a blood transfusion every three to four weeks to maintain hemoglobin levels.
Beti-cel is a one-time gene therapy designed to treat the underlying cause of transfusion-dependent β-thalassemia.
bluebird bio based its biologics license application submission on data from the Phase 3 HGB-207 and HGB-212 studies and the Phase 1/2 HGB-204 and HGB-205 studies. Together, these studies represent over 220 patient years of experience with beti-cel.
The results of the studies included 63 pediatric, adolescent, and adult patients treated with beti-cel across β0/β0 and non-β0/β0 genotypes. The data included two patients with at least seven years of follow-up, eight with at least six years of follow-up, and 19 with at least five years of follow-up.
Across all Phase 3 studies of the therapy, 89 percent of patients across all ages and genotypes achieved transfusion dependence.
