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Rotercept (Luspatercept) is a first-in-class erythroid maturation agent. It is an artificial soluble recombinant fusion protein, composed of the Fc domain of human immunoglobulin G1 (IgG1) and the extracellular domain of activin receptor type IIB (ActRIIB). Its mechanism of action involves regulating late-stage erythroid maturation and improving ineffective erythropoiesis by binding to select TGF-β superfamily ligands, thereby reducing aberrant Smad2/3 signaling. Rotercept was initially developed by Celgene and Acceleron, and later acquired by Bristol-Myers Squibb (BMS). It is approved for the treatment of anemia in adult patients with transfusion-dependent β-thalassemia and for anemia associated with myelodysplastic syndromes (MDS) with ring sideroblasts.
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