By Fineline Cube US pharmaceutical giant Bristol-Myers Squibb (BMS; NYSE: BMY) has announced the commercial launch of its first-in-class erythrocyte maturation agent, Reblozyl (luspatercept), in China. The product, filed by its subsidiary Celgene, received approval in China in January 2022 for the treatment of adult β-thalassemia patients who require regular red blood cell transfusions of ≤ 15 units per 24 weeks.
Understanding β-Thalassemia
β-thalassemia is a hereditary blood disorder characterized by anemia due to decreased or absent synthesis of the globin peptide chain. Traditional clinical treatment strategies primarily involve blood transfusions and iron-chelation therapy. Reblozyl, a novel fusion protein, works by binding to the TGF-β superfamily ligand, a key cytokine that regulates red blood cell maturation. This action reduces the abnormally enhanced Smad 2/3 signal transduction, thereby promoting late-stage red blood cell maturation and enabling the body to produce more mature red blood cells.
Global Approvals and Development
Reblozyl, co-developed with Acceleron Pharma Inc., has obtained two indication approvals worldwide. The first is for adult β-thalassemia patients requiring regular red blood cell transfusions. The second is for the treatment of very low- to moderate-risk adult patients with ring sideroblasts myelodysplastic syndromes (MDS-RS) who have failed erythropoiesis stimulating agent (ESA) therapy and require transfusion of two or more units of red blood cells within eight weeks, or for anemia in adult patients with myelodysplastic syndrome/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).-Fineline Info & Tech