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Zynteglo, New gene therapy to treat rare inherited blood condition

EMA has recommended granting a marketing authorisation in the European Union for a genetically modified product for beta-thalassaemia, a rare inherited blood condition that causes severe anaemia. Zynteglo is intended for adult and adolescent patients 12 years and older who need regular blood transfusions to manage their disease and have no matching donor for a stem cell transplant.

The active substance of Zynteglo is an autologous CD34+ cell enriched population that contains hematopoietic stem cells (HSC) transduced with lentiviral vector (LVV) encoding the βA‑T87Q-globin gene. Zynteglo is a gene therapy medicinal product that adds functional copies of a modified β-globin gene into the patients’ haematopoietic stem cells through transduction of autologous CD34+ cells with BB305 lentivirus vector, thereby addressing the underlying genetic cause of the disease.

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.

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.

Zynteglo was designated as an orphan medicinal product on 24 January 2013. During the development protocol assistance was received on multiple occasions to obtain guidance on the quality aspects and the clinical study programme, including the demonstration of significant benefit.

The opinion adopted by the CHMP is an intermediary step on Zynteglo’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once the marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

The applicant for Zynteglo is bluebird bio (Netherlands) B.V.

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