FDA Expands Approval of Reblozyl to Treat Anemia in Adults with MDS

Rare Daily Staff

The U.S. Food and Drug Administration expanded the approval for Bristol Myers Squibb and Acceleron Pharma’s Reblozyl to treat myelodysplastic patients with anemia.

Reblozyl is the first and only erythroid maturation agent (EMA), for the treatment of anemia failing an erythropoiesis stimulating agent and requiring two or more red blood cell units over eight 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). Ring sideroblasts are precursors to mature red blood cells with iron-loaded mitochondria.

Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Myelodysplastic syndromes (MDS) are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells, and platelets, which can lead to anemia and frequent or severe infections. People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions, and infections.

Reblozyl promotes late-stage red blood cell maturation in animal models. The FDA first approved Reblozyl in November 2019 for the treatment of anemia in adults with beta thalassemia who require regular red blood cell transfusions.

The FDA approval in MDS is based on results from the pivotal Phase 3 MEDALIST trial, a randomized, double blind, placebo-controlled, multi-center study evaluating the efficacy and safety of Reblozyl in patients with IPSS-R-defined very low-, low- and intermediate-risk non-del(5q) myelodysplastic syndromes (MDS) with ring sideroblasts. All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) therapy or were ESA naïve and unlikely to respond due to endogenous serum erythropoietin ≥200 U/L, and had no prior treatment with disease modifying agents.

In the trial, a significantly greater proportion of patients receiving Reblozyl achieved independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared with those receiving placebo, meeting the study’s primary endpoint. Additionally, a significantly greater proportion of patients receiving Reblozyl vs. placebo achieved at least 12 weeks of independence from transfusions within the first 24 and 48 weeks of the study. Results from MEDALIST were published in the New England Journal of Medicine in January 2020.

“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, professor and chief of section of myelodysplastic syndromes in the 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.”

Photo: Guillermo Garcia-Manero professor and chief of section of myelodysplastic syndromes in the department of leukemia, University of Texas MD Anderson Cancer Center.