luspatercept   Click here for help

GtoPdb Ligand ID: 10538

Synonyms: ACE-536 | ACE536 | luspatercept-aamt | Reblozyl®
Approved drug
luspatercept is an approved drug (FDA (2019), EMA (2020))
Compound class: Peptide
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]. Mechanistically luspatercept acts as a ligand trap for growth/differentiation factor-11 (GDF11; and other Smad2/3-activating ligands), which reduces GDF11-induced Smad2/3 activation, and blocks the inhibitory effect of GDF11 on erythroid precursor maturation. Functionally, it promotes maturation of late-stage erythroid precursors, which is in contrast to erythropoietin, which stimulates proliferation of early-stage erythrocyte precursors. Using Gdf11 knockout, Guerra et al. (2019) dispute GDF11 as the molecular target of luspatercept and its proposed inhibitory effect on late erythropoiesis [1]. They do not propose an alternative mechanism of action.
Classification Click here for help
Compound class Peptide
Approved drug? Yes (FDA (2019), EMA (2020))
International Nonproprietary Names Click here for help
INN number INN
9823 luspatercept
Synonyms Click here for help
ACE-536 | ACE536 | luspatercept-aamt | Reblozyl®
Database Links Click here for help
Specialist databases
IMGT/mAb-DB 487
Other databases
DrugBank Ligand DB12281
GtoPdb PubChem SID 387065620
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