ZYNTEGLO Gene Therapy Launches in Germany for Treatment of Beta Thalassemia

January 27, 2020 – Earlier this month, bluebird bio announced the launch in Germany of ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene), the first gene therapy approved in Europe for the treatment of transfusion-dependent beta thalassemia. ZYNTEGLO is a one-time gene therapy for patients 12 and older with transfusion-dependent beta-thalassemia who do nothave a B0/B0 genotype, for whom hematopoietic stem cell transplantation is appropriate but for whom an HLA-matched donor is not available. This is the first time that ZYNTEGLO is commercially available anywhere.

ZYNTEGLO addresses the underlying genetic cause of transfusion-dependent beta thalassemia, and offers patients the potential to become transfusion independent, which is expected to be lifelong. bluebird bio is working to create qualified treatment centers that will administer ZYNTEGLO, and has established a collaboration with University Hospital of Heidelberg as the first qualified treatment center in Germany.

“For patients with TDT, lifelong chronic blood transfusions are required in order to survive. We are thrilled to announce that ZYNTEGLO will now be available for patients in the EU living with this severe disease,” says Alison Finger, bluebird bio’s chief commercial officer. “In addition to confirming manufacturing readiness of our partner, apceth Biopharma GmbH, bluebird has also submitted a dossier to the Joint Federal Committee (G-BA) in Germany for drug benefit assessment. We would like to thank our collaborators for their commitment in helping us transform the healthcare system by accepting innovative payment models, and we look forward to treating our first commercial patient soon.”

As for the United States, the Food and Drug Administration (FDA) granted LentiGlobin for β-thalassemia (the US name for ZYNTEGLO) Orphan Drug status and Breakthrough Therapy designation for the treatment of beta transfusion-dependent beta thalassemia. LentiGlobin for β-thalassemia is not currently approved in the United States. bluebird bio has initiated the application submission process for FDA approval in the US, and plans to complete the process in the first half of 2020.

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