bluebird bio has made Zynteglo commercially available for the first time after the one-time gene therapy was launched in Germany.
The therapy is intended for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype.
TDT is a severe genetic disease caused by mutations in the β-globin gene that result in significantly reduced or absent adult haemoglobin (HbA).
In order to survive, people with TDT maintain haemoglobin (Hb) levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.
Zynteglo is a one-time gene therapy that addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent, which, once achieved, is expected to be lifelong.
Due to the highly technical and specialized nature of administering gene therapy in rare diseases, bluebird bio is working with institutions that have expertise in stem cell transplant as well as in treating patients with TDT to create qualified treatment centres that will administer Zynteglo.
bluebird bio has established a collaboration with University Hospital of Heidelberg as the first qualified treatment centre in Germany.
In addition, the company has entered into value-based payment agreements with multiple statutory health insurances in Germany to help ensure patients and their healthcare providers have access to Zynteglo and that payers only pay if the therapy delivers on its promise.