A new and encouraging chapter is finally opening up for patients with thalassaemia, following the recent approval of the US Food and Drug Administration (FDA) to market the drug Reblozyl, which aims to help patients with beta-thalassaemia major become more independent from regular, and often painful, blood transfusions.
This important development is coming to overturn the existing data in the management – and probably very soon, in the treatment – of thalassaemia, and reflects the rapid scientific progress recorded in the field of the disease’s research in recent years. It is worth noting that this is the first time that a new generation medication is approved by a world-class drug licensing regulator.
To date, the main option that allows patients with β-thalassaemia to manage their condition are lifelong blood transfusions, which over time lead the body to iron overload, with potentially life-threatening consequences, and hence the necessary systematic use of chelating agents.
The new revolutionary treatment has reduced by at least 33% the number of blood transfusions needed over 3 months in a noteworthy number of patients transfused during the final clinical trials. Over time, the reduction in specific patient groups has even exceeded 50%. As a result, the improvement of the patients’ quality of life, through the extension of the interval between blood transfusions, is enormous.
Given the fact that Reblozyl is also soon expected to be licensed in Europe by the European Medicines Agency (EMA), the Thalassaemia International Federation (TIF), a leader in informing and educating organized patient groups and health professionals active in the field of haemoglobinopathies, intends to organize very soon an Informative Meeting with prominent physicians, and representatives of patient organisations and the Cypriot Ministry of Health, with the aim of launching an early dialogue on the marketing, costs and patients’ access to this new treatment.