luspatercept

Luspatercept is a recombinant fusion protein and erythroid maturation agent used to treat anemia in adults with beta thalassemia who require regular red blood cell transfusions, as well as certain types of myelodysplastic syndromes (MDS) in adults who are transfusion-dependent or have not responded to, or cannot receive, erythropoiesis-stimulating agents. Luspatercept consists of a modified extracellular domain of the human activin receptor type IIB fused to the Fc domain of human IgG1. It acts as a ligand trap for select transforming growth factor-beta (TGF-β) superfamily ligands, reducing Smad2/3 signaling and promoting late-stage erythroid maturation. This mechanism alleviates ineffective erythropoiesis and increases red blood cell production. It was originally developed by Acceleron Pharma in collaboration with Celgene; both companies were later acquired by Bristol Myers Squibb. The precise TGF-beta superfamily ligands targeted by luspatercept are not fully defined; GDF11 has been excluded as a primary functional target. Luspatercept is considered first-in-class for its mechanism and indication.

Brand names

Reblozyl

Other names

luspatercept-aamt