Bluebird Bio’s one-time gene therapy, Zynteglo, has been cleared by EU regulators for the treatment of beta thalassemia in patients aged 12 and older. Zynteglo is a LentiGlobin therapy, through which mobilized autologous CD34-positive cells are obtained from patients and transduced ex vivo with the LentiGlobin BB305 vector, which encodes adult hemoglobin (HbAT87Q). Patients undergo conditioning with busulfan, then modified cells are re-infused.
The single-dose treatment, which aims to eliminate or substantially reduce a patient’s need for blood transfusions, will initially be administered to 2,500 patients across Germany, Italy, the U.K., and France.
In a value- and outcome-based payment model, Bluebird Bio set the price for the gene therapy at €1.575 million, or approximately $1.78 million. This amount is payable as installments of €315,000 ($356,600) over five years, and payments are only made if the treatment proves successful.
U.S. approval of Zynteglo for treatment of beta thalassemia is anticipated in 2020; meanwhile, Bluebird Bio continues to develop the therapy for the treatment of sickle cell anemia.