FDA Approves Bluebird Bio’s Gene Therapy For Rare Blood Disease

The U.S. Food and Drug Administration approved bluebird bio Inc.’s (BLUE) Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

BLUE closed Wednesday regular trading at $6.78 up $0.20 or 3.04%. In the after-hours trade, the stock further gained $0.47 or 6.93%.

Zynteglo is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin, a hemoglobin component.

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood, through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications.

Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires life-long red blood cell transfusions as the standard course of treatment. The regular transfusions can be associated with multiple health complications of their own, including problems in the heart, liver and other organs due to an excessive build-up of iron in the body.

Zynteglo was reviewed under Priority Review, and the company received a priority review voucher upon approval. Zynteglo was previously granted Orphan Drug designation and Breakthrough Therapy designation.

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