EU approves first red blood cell maturant Reblozyl to treat blood transfusion-dependent anemia

The European Commission has approved The BMS's Reblozyl (luspatercept) for the treatment of blood transfusion-dependent anemia associated with marrow hyperplasia syndrome (MDS) or beta thalassemiaThe approval makes Reblozyl the first and only red blood cell maturation agent approved by the European UnionPhoto Credit:Reblozyl is a soluble fusion protein that is a ligand trap made from the fc domain of humanionateg1 (IgG1) and the extracellular domain of actRIIB receptors By targeting the binding, the specific ligands of the late RBC mature conversion growth factor (TGF)-beta superfamily can be adjusted to reduce the activation of Smad2/3 signaling pathways, improve the production of ineffective red blood cells, and thus promote the differentiation and maturation of late-stage red blood cells, and improve hemoglobin levelsReblozyl's approval was based on the results of the Phase III MEDALIST study, which showed that Reblozyl significantly improved patient dependence on red blood cell (RBC) blood transfusionsThe proportion of patients who were at least 8 weeks without RBC transfusions during the first 24 weeks of the trial received 37.9 percent of Reblozyl treatment, compared with 13.2 percent of those who took a placeboThus reached the main end point of the experimentthe trial also reached a secondary endpoint of at least 12 weeks without dependence on blood transfusions during the first 24 and 48 weeks of the study, with significantly higher rates of patients receiving Reblozyl than placebosmost common adverse reactions at all levels include fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity, hypertension, headache, upper respiratory tractinfections, bronchitis and urinary tract infections