luspatercept

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luspatercept

GtoPdb Ligand ID: 10538

Synonyms: ACE-536 | ACE536 | luspatercept-aamt | Reblozyl®

luspatercept is an approved drug (FDA (2019), EMA (2020))

Comment: Luspatercept (ACE-536) is a fusion protein comprising human ACVR2B (activin receptor type 2B) extracellular domain (which is mutated to reduce activin binding), fused with human immunoglobulin G1 Fc fragment [2]. Luspatercept was initially proposed as a ligand trap for growth/differentiation factor-11 (GDF11; and other TGFβ family ligands). Mechanistically, it reduces Smad2/3 activation, and promotes maturation of late-stage erythroid precursors, independently of the erythropoietin (EPO) pathway. In contrast, erythropoietin stimulates proliferation of early-stage erythrocyte precursors.
Using Gdf11 knockout, Guerra et al. (2019) disputed GDF11 as an important molecular target of luspatercept and its proposed inhibitory effect on late erythropoiesis [1].

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Summary of Clinical Use
Luspatercept (ACE-536) is used as an erythroid maturation agent. It was granted FDA approval in November 2019 as a treatment for adult patients with beta thalassemia-associated anemia who require regular red blood cell transfusions. European Union approval by the EMA was issued in June 2020, indicated for the treatment of anemia associated with beta thalassemia and myelodysplastic syndromes.

Summary of Clinical Use

Luspatercept (ACE-536) is used as an erythroid maturation agent. It was granted FDA approval in November 2019 as a treatment for adult patients with beta thalassemia-associated anemia who require regular red blood cell transfusions. European Union approval by the EMA was issued in June 2020, indicated for the treatment of anemia associated with beta thalassemia and myelodysplastic syndromes.