Luspatercept (Reblozyl)
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Mechanism of action
From the NCI Drug Dictionary: A soluble, recombinant fusion protein composed of a modified form of the extracellular domain of human activin receptor type IIb (ActRIIb) and linked to the human IgG1 Fc domain, with red blood cell stimulating activity. Upon subcutaneous administration, luspatercept inhibits several ligands in the transforming growth factor (TGF)-beta superfamily. This prevents activation of a variety of TGF-beta superfamily members involved in late stage erythropoiesis and results in an increased differentiation and proliferation of erythroid progenitors. Luspatercept acts at a different, later stage than erythropoietin.
Diseases for which it is used
History of changes in FDA indication
- 2019-11-08: Initial FDA approval for adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. (Based on BELIEVE)
- 2020-04-03: Approved for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). (Based on MEDALIST)
History of changes in EMA indication
- 2020-06-25: Initial authorization as Reblozyl. Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.
- 2020-06-25: Initial authorization as Reblozyl. Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia associated with beta thalassaemia.
- 2023-02-27: Extension of indication in beta thalassaemia to include adult patients with non-transfusion dependent beta-thalassaemia (NTDT).
Also known as
- Code name: ACE-536
- Generic name: luspatercept-aamt
- Brand name: Reblozyl