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EMA approves Bluebird Bio's gene therapy Zynteglo

Published on 04/06/19 at 09:15am

The European Commission has granted a conditional marketing authorisation to bluebird bio’s gene therapy Zynteglo.

The Massachusetts-based firm’s one time treatment has been approved for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT).

In order to survive, patients with TDT must maintain their haemoglobin levels through regular blood transfusions.  These transfusions carry the risk of progressive, multi-organ damage which is caused by iron overload.

Zynteglo, which addresses the underlying causes of TDT, offers patients the potential to live their lives without being dependant on blood transfusions.

Andrew Obenshain, bluebird bio European General Manager commented: “EC authorisation of Zynteglo brings a new treatment option, in the form of the first one-time gene therapy for a subset of TDT patients one step closer to being available to patients across Europe.”

“This is an important time to acknowledge the support of the TDT community, patients and clinical investigators in helping us to get to where we are today. We will now be prioritising reimbursement, ensuring that we work with national payer agencies in each country to enable access to this treatment across Europe”

Dr Franco Locatelli, Professor of Pediatrics, Sapienza University of Rome, Italy and Director, Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, added: “As one of the investigators in the clinical studies of Zynteglo, I have witnessed first-hand the hope this gene therapy can provide to patients and their families who have often been managing this disease and transfusions for years, often for decades.”

“This authorisation by the European Commission means we now have a gene therapy for certain patients with TDT that has the potential to transform lives by offering the possibility of a transfusion-free future.”

Louis Goss

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