EMA has recommended granting a marketing authorisation in the EU for a genetically modified product for beta-thalassaemia, a rare inherited blood condition that causes severe anaemia. Zynteglo is Bluebird Bio’s first gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who need regular blood transfusions to manage their disease and have no matching donor for a stem cell transplant.
Patients suffering from beta-thalassaemia cannot produce enough beta-globin, a key component of haemoglobin, the protein that carries the oxygen in the blood from the lungs to the rest of the body. As a consequence, they have far fewer red blood cells than normal and suffer from chronic severe anaemia.
These patients often require lifelong blood transfusions for their anaemia. Chronic transfusions can cause iron overload, hence patients need to take medication to remove excess iron. So far, the only curative treatment option is stem cell transplantation from a healthy donor. However, in the absence of a matched family donor, finding appropriate donors is often difficult and stem cell transplantation with unrelated donors is associated with severe side-effects and reactions. Patients who depend on blood transfusions to manage beta-thalassaemia therefore have an unmet medical need for new treatments.
Zynteglo is a new therapeutic option for patients for whom a related donor for stem cell transplantation is not available. Using a lentiviral viral vector, it adds functional copies of a modified β-globin gene into a patient’s own stem cells, thereby addressing the underlying genetic cause of the disease.
“The positive opinion for ZYNTEGLO by the Committee for Medicinal Products for Human Use (CHMP) is a crucial step toward providing what would be the first one-time gene therapy for people living with TDT. We look forward to the upcoming decision from the European Commission”, said David Davidson, M.D., Bluebird’s Chief medical officer.
Zynteglo should only be administered in a qualified treatment centre by a physician with experience in stem cell transplantation and in the treatment of patients with beta-thalassaemia.
In the two main studies to demonstrate the effects of Zynteglo it was shown that the majority of patients who do not have a β0/β0 genotype treated with Zynteglo no longer needed regular blood transfusions.
The most common side effects were thrombocytopenia (low blood platelet count), abdominal pain, non-cardiac chest pain, pain in the extremities, dyspnoea and hot flush.
A final decision by the EC for ZYNTEGLO is anticipated in the second quarter of 2019.