Bluebird Bio Receives FDA Breakthrough Therapy Designation For Lentiglobin Drug Therapy

Today Bluebird Bio (BLUE) announced that it has received FDA breakthrough therapy designation for its drug therapy known as LentiGlobin used to treat a major disease known as beta-thalassemia major. LentiGlobin BB305 is used to treat both beta-thalassemia major and severe sickle cell diseases. The FDA Breakthrough Therapy designation is given to companies to expedite drugs that have clinical benefits over other current treatments. What LentiGlobin may ultimately do is allow patients to not have to rely on transfusions for their disease. 

Beta-thalassemia is a disease whereby the patient's body produces lower counts of beta hemoglobins. Hemoglobins are the proteins responsible for carrying oxygen through the red blood cells. Beta-thalassemia major is the severe form of this anemia because this disease causes the patient to have two genes for beta-thalassemia and no normal beta-chain genes. This is more severe than beta-thalessemia minor and is a severe illness. Problems that arise from beta-thalessemia major are slower than normal growth rate, fatigue, and reduced bone marrow in the body. As mentioned above, beta-thalassemia major patients require lifelong blood transfusions. The problem with lifelong blood transfusions is that they lead to iron overload in the body, which in turn may lead to death. 

Therefore a therapy like Bluebird Bio's, utilizing gene therapy with LentiGlobin, may help stop the need for these  transfusions. Bluebird Bio reported initial results from 4 patients who took the LentiGlobin therapy. The results were that these 4 patients' bodies were able to produce enough hemoglobin on their own and eliminated the need for these patients to undergo any kind of blood transfusion whatsoever. There is still a lot of work to be done for further results but these early results were remarkable and is probably why the FDA granted breakthrough therapy designation to LentiGlobin.