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Reblozyl
Published
Product name
Reblozyl
Active ingredient
Luspatercept
Submission type
New chemical entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Reblozyl luspatercept was approved for the following therapeutic use:
Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia (requiring 2 or more RBC units over 8 weeks) due to very low, low and intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS).
Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia associated with beta thalassaemia.
Limitation of Use
Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anaemia.
How this medicine works
Luspatercept is a recombinant fusion protein consisting of a modified form of the extracellular domain of the human activin receptor type IIB (ActRIIB) linked to the human immunoglobin G1 (IgG1) fragment constant (Fc) domain. It acts as a ligand trap for select transforming growth factor beta (TGF-β) superfamily ligands. By binding to certain endogenous ligands that act as negative regulators of erythropoiesis, luspatercept inhibits downstream Smad2/3 signalling, resulting in erythroid maturation through differentiation of late-stage erythroid precursors (normoblasts) in the bone marrow. TGF-β superfamily signalling through Smad2/3 is abnormally high in disease models characterised by ineffective erythropoiesis, that is myelodysplastic syndromes (MDS) and β-thalassaemia, and in the bone marrow of MDS patients.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Reblozyl was considered favourable for the therapeutic use approved.
Sponsor