Beta-Thalassemia Major Treatment Designated Breakthrough Therapy
bluebird bio announced that the FDA has granted Breakthrough Therapy designation to LentiGlobin BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major.
The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. In four of the eight patients, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions.
LentiGlobin BB305 Drug Product is being developed to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient's own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation.
For more information visit bluebirdbio.com.