Bristol Myers Squibb and Acceleron Pharma Inc. announced the U.S. Food and Drug Administration (FDA) has approved Reblozyl® (luspatercept-aamt), the first and only erythroid maturation agent (EMA), 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). Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.
“In clinical trials, Reblozyl has shown to have significant benefit for the treatment of anemia in patients with myelodysplastic syndromes who have ring sideroblasts,” said Guillermo Garcia-Manero, M.D., professor and chief of Section of Myelodysplastic Syndromes, Department of Leukemia, University of Texas MD Anderson Cancer Center. “Anemia is a serious consequence of MDS, requiring the majority of these patients to receive regular red blood cell transfusions, which can lead to additional complications, such as iron overload, transfusion site reactions and infections. In our current environment, we are reminded of the significant burden frequent blood transfusions can have on individuals and the healthcare system.”
As a potential blockbuster drug touted by Bristol Myers Squibb during its Celgene buyout, Reblozyl’s future success was in large part dependent on an approval for a group of bone marrow disorders called myelodysplastic syndromes (MDS). Now, it has that nod.
Reblozyl was among five drugs in Celgene’s late-stage pipeline that it said could potentially reach blockbuster status. The other four are JAK inhibitor Inrebic, which the FDA cleared for myelofibrosis in August; just-approved multiple sclerosis drug Zeposia; as well as forthcoming CAR-T therapies liso-cel and ide-cel.