Green light to Bluebird’s gene therapy in Europe

Biotech Bluebird has announced today that EMA has accepted the Marketing Authorization Application (MAA) for its LentiGlobin-based  investigational treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.

The authorization reflects the excellent clinical data delivered by the drug, especially in the Phase-III Northstar-2 (HGB-207) clinical trial. TDT is an hereditary blood disorder, caused by a mutation in the β-globin gene, which causes ineffective red blood cell production leading to severe anemia.

(Source: Bluebird)