Reblozyl’s long-awaited approval in Europe has finally arrived!
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 address the ineffective erythropoiesis associated with β-thalassaemia and MDS, decrease patients’ dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients, and is the first and only erythroid maturation agent to be approved in the European Union (EU), representing a new class of therapy. The approval was based on positive data from the phase III MEDALIST and BELIEVE studies, which evaluated the ability of Reblozyl to effectively address anemia associated with β-thalassaemia and MDS, respectively.
Approximately, 25 million blood transfusions occur every year in the EU, some of which are needed by patients with anemia due to hematologic diseases, whilst the drug is already approved in the United States for the treatment of anaemia in adult patients with beta thalassaemia who require regular Red Blood Cells (RBCs) transfusions.
“While beta thalassaemia remains an orphan disease, the lifelong blood transfusions often needed by patients can have a significant impact on the limited blood supply in their communities, and there are few treatment alternatives,” said Maria Domenica Cappellini, M.D., lead investigator of the BELIEVE study, Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda. “The European Commission’s approval of Reblozyl provides eligible adult patients with beta thalassemia a new, much needed treatment option for their anemia, and with it, the possibility of becoming less dependent on red blood cell transfusions.”
Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is already approved in the U.S. for the treatment of:
- anaemia in adult patients with beta thalassaemia who require regular RBCs transfusions, and
- anaemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anaemia.