Gene therapy Zynteglo for TDT in Europe

August 6, 2019 0 By FM

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.