bluebird bio, Inc. announced the US Food and Drug Administration (FDA) has approved ZYNTEGLO, a one-time gene therapy, which is custom-designed to treat the underlying genetic cause of the beta-thalassemia in the adult and pediatric patients who now require regular red blood cell (RBC) transfusions.
Andrew Obenshain, chief executive officer of bluebird bio, said this US FDA approval of ZYNTEGLO offers patient with beta-thalassemia the possibility of freedom from the regular red blood cell transfusions, and iron chelation, with unlocking new possibilities in their daily lives.
Andrew Obenshain, CEO of bluebird bio, said after over a decade of research and clinical development, and also through the perseverance of patients, clinicians, and their families, this FDA approval marks a watershed moment for the field of gene therapy.
Beta-thalassemia is a rare, genetic blood disease, which is caused by mutations in the beta-globin gene, and is characterized by significantly decreased adult haemoglobin production. The patients with the more serious form, and experience serious anemia and lifelong dependence on the regular red blood cell transfusions, which is a lengthy process that patients typically undergo in every 2 to 5 weeks.
Despite the advances in treatment and the improved transfusion techniques, which temporarily address symptoms of anemia and people with beta-thalassemia, who now require regular transfusions now have an increased risk for mortality and mobidity because of complications from iron overload related to treatment.
