Like every year, the International Thalassaemia Day this year has a distinct theme, aiming to achieve its mission in enhancing lives and exceeding expectations for patients living with thalassaemia across the world.
The 2020 theme is:
‘’The dawning of a new era for thalassaemia:
Time for a global effort to make novel therapies accessible and affordable to patients ».
Read the ITD 2020 Full Press Release HERE.
Why was this theme selected:
The remarkable scientific developments in the field of thalassaemia, in current years, including the recent approval by official regulatory authorities of new therapies that promise to change dramatically the course of this disease, have inspired the selection of this topic for the International Thalassaemia Day 2020. Here at TIF we believe that these new and innovative therapies are marking a turning point in the history of the disorder, and offer extraordinary opportunities, which we must seize if we are to improve our quality of life.
Furthermore, this theme corresponds with the Federation’s recent decision to launch the “TIF’s International Forum on ACCessibility, Effectiveness and Sustainability of TherapieS for Haemoglobin Disorders (TIF.ACCESS)”, which with the support of TIF’s International Scientific Advisory Panel and Patient Community Advisory Board, aims to:
- provide timely and trustworthy information and training to patients, healthcare professionals and other stakeholders and partners active in the field of haemoglobinopathies, considering the upsurge of innovative therapies, and
- address issues related to the Accessibility, Safety, Effectiveness and Availability (ASEA) of novel therapies in different regions of the world, taking into account each affected country’s Human Development Index and Health Spending.
New & innovative therapies for thalassaemia:
The discovery and development of novel therapies and drugs for thalassaemia has been remarkable, to say the least, during the past few years. In 2019, various pharmaceutical companies have achieved significant progress in clinical trials on medicinal products for this haemoglobin disorders, 2 of which stand out given their recent marketing authorization by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). In the meantime, other therapies and drugs for thalassaemia with significant potential are currently in the pipeline.
ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene†) is the first and only one-time groundbreaking gene therapy for transfusion dependent β-thalassaemia by Bluebird Bio that gives patients the potential to reach complete transfusion independence. The therapy was granted approval by the EMA in June 2019 and Germany is the first country to commercially offer Zynteglo with the University Hospital of Heidelberg being the first qualified treatment center to administer this gene therapy to patients.Learn more about ZYNTEGLO
TIF has issued 2 educational brochures about the revolutionary gene therapy which can help you improve your understanding on its application for patients with thalassaemia.
Click on the images to discover more:
In November 2019, the FDA approved REBLOZYL (Luspatercept-aamt) for the treatment of anaemia in β-thalassaemia in adults needing regular red blood cell (RBC) transfusions. Reblozyl is an erythroid maturation agent, which was co-developed by Celgene and Acceleron Pharma. Approval from the EM is currently pending and expected to be obtained in 2020. This drug promises immense improvements to the quality of life and health of thalassaemia patients in addition to significant wider public health benefits, such as health expenditure.