Innovative Therapies

Bristol Myers Squibb Canada (BMS) and Acceleron Pharma Inc. announced today that Health Canada has approved REBLOZYL® (luspatercept) for the treatment of adult patients with red blood cell (RBC) transfusion-dependent anemia…

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…

IMARA Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of haemoglobin, announced the U.S. Food and Drug…

This Annual Report showcases the Federation’s intensive efforts that took place in 2019 with the aim to ensure equal access to quality healthcare for every patient with thalassaemia and haemoglobin…

Novartis announced last Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (crizanlizumab)…

The swift scientific progress made in the field of thalassaemia research has gained momentum with the market authorisations of both the Zynteglo Gene Therapy by the European Medicines Agency (EMA)…

Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease…

IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the…

Bristol Myers Squibb (BMS) & Acceleron Pharma announced today that the European Commission approved Reblozyl (luspatercept) for the treatment of Transfusion-Dependent β-#Thalassaemia (TDT) in adult patients. Reblozyl has the potential to…