U.S. FDA approves bluebird bio's gene therapy for rare blood disorder

The U.S. Food and Drug Administration on Wednesday approved bluebird bio's gene therapy for the treatment of a rare blood disorder. The approval allows the use of beti-cel, to be branded as Zynteglo, as a one-time treatment for pediatric and adult beta-thalassemia patients dependent on regular transfusions. Treatment for people with beta-thalassemia often consists of lifelong regimens of chronic blood transfusions.

The U.S. Food and Drug Administration on Wednesday approved bluebird bio's gene therapy for the treatment of a rare blood disorder.

The approval allows the use of beti-cel, to be branded as Zynteglo, as a one-time treatment for pediatric and adult beta-thalassemia patients dependent on regular transfusions. Bluebird bio's shares were halted prior to the decision.

Beta-thalassemia is an inherited disorder caused by beta-globin gene mutations, which reduce the production of hemoglobin. Treatment for people with beta-thalassemia often consists of lifelong regimens of chronic blood transfusions. Beti-cel works by adding functional copies of a modified form of beta-globin in a patient's stem cells in order to correct the hemoglobin deficiency.

The agency's approval was largely expected after the drug received unanimous endorsement from a panel of outside advisers to the FDA in June.