bluebird bio, Inc. announced that the European Medicines Agency (EMA) approved the refined commercial drug product manufacturing specifications for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
“We look forward to serving TDT patients with ZYNTEGLO and providing a treatment option that offers the possibility of a transfusion-free future,” said Alison Finger, chief commercial officer, bluebird bio. “This is one step along the commercial journey as we advance our ongoing launch and market access activities on a country-by-country basis, with the goal of enrolling our first commercial patient in 2019.”
The refined commercial drug product specifications support the efficacy and safety profile of ZYNTEGLO and will give patients the best opportunity for clinically meaningful outcomes consistent with the results that were foundational to the conditional marketing authorization in the European Union. ZYNTEGLO addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent, which once achieved is expected to be life-long.