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Luspatercept Injection [Reblozyl]

Groupe Francophone des Myelodysplasies · Phase 2 active Small molecule

Luspatercept is a recombinant fusion protein that acts as a ligand trap for members of the TGF-β superfamily, modulating Smad2/3 signaling to enhance erythroid maturation.

Luspatercept is a recombinant fusion protein that acts as a ligand trap for members of the TGF-β superfamily, modulating Smad2/3 signaling to enhance erythroid maturation. Used for Myelodysplastic syndromes (MDS) with ring sideroblasts, Beta-thalassemia.

At a glance

Generic nameLuspatercept Injection [Reblozyl]
Also known asACE-536
SponsorGroupe Francophone des Myelodysplasies
Drug classErythroid maturation agent
TargetTGF-β superfamily ligands (GDF11, activin A)
ModalitySmall molecule
Therapeutic areaOncology
PhasePhase 2

Mechanism of action

By binding to growth differentiation factor 11 (GDF11) and activin A, luspatercept prevents their interaction with the activin type II receptor, thereby reducing inhibitory signals in the bone marrow and promoting the maturation of red blood cells.

Approved indications

Common side effects

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