The European Commission granted conditional marketing authorization to Bluebird Bio's Lentiglobin BB305 gene therapy (ZYNTEGLOTM) for transfusion-dependent β-thalassaemia (TDT). ZYNTEGLOTM is a one-time gene therapy (autologous CD34+ cells encoding βA-T87Q-globin gene) for a specific genotype of TDT, cleared for use in patients aged 12 and older who are able to receive a stem cell transplant but have no matched donor. These are exciting news as ZYNTEGLOTM is the first gene therapy to gain regulatory approval for TDT in the European Union, offering for the first time the possibility of a transfusion-free future and a better quality of life to severely-affected patients. The company will offer the therapy first in Germany, followed by Italy, the U.K. and France, and hopes to launch ZYNTEGLOTM in the U.S. next year.
More information: Bluebird Bio press release, EMA Medicine Report