Bluebird Hangs $1.8 Million Price Tag on Zynteglo
Bluebird Bio has set a high price in Europe for its gene therapy for a rare blood disease, pricing Zynteglo at $1.8 million.
The European Commission handed Zynteglo (autologous CD34+ cells encoding BA-T87Q-globin gene) conditional marketing approval on June 3 for patients aged 12 years and older that suffer from a rare form of anemia, transfusion-dependent B-thalassemia.
Patients with TDT must receive blood transfusions for life, which puts them at risk for organ failure because of iron overload. The approval was supported by data from a Phase 1/2 trial, which showed three of the four patients were able to go without a transfusion for at least a year after taking the drug.