Gene therapy Zynteglo for TDT in EuropeAugust 6, 2019
Bluebird Bio has received the European Commission’s conditional marketing authorisation for its groundbreaking gene therapy Zynteglo (autologous CD34+ cells encoding ßA-T87Q-globin gene).
The one-time gene therapy has been approved for patients 12 years and older with transfusion-dependent ß-thalassaemia (TDT).
This first conditional approval is in the moderately severe TDT form of the disease for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
A number of small studies have shown the drug frees a majority of patients from the need to have regular blood transfusions.
Though the company is yet to disclose its price, analysts expect it to be anywhere between €450,000 and €1 million ($500,000 – $1.12m)
Meanwhile, reports indicate the launch of the highly anticipated, and very expensive gene therapy will not occur until 2020 due to certain manufacturing concerns.