FDA Approves Reblozyl for Anemia in Adults with Lower-Risk Myelodysplastic Syndromes

April 4, 2020

The Food and Drug Administration approved the first and only erythroid maturation agent to treat patients with MDS who require red blood cell transfusions and have failed an erythropoiesis stimulating agent.

The Food and Drug Administration (FDA) approved Reblozyl (luspatercept-aamt)

to treat patients with myelodysplastic syndromes (MDS) who require red blood cell transfusions and have failed an erythropoiesis stimulating agent, according to Bristol Meyers Squibb, the agent’s manufacturer.

With this approval, Reblozyl is the first and only erythroid maturation agent approved for this indication and is the first new treatment option in over a decade for these patients.

"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,” Dr. Guillermo Garcia-Manero, professor and chief of the Section of Myelodysplastic Syndromes in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, in Houston, said in a press release.

“In our current environment, we are reminded of the significant burden frequent blood transfusions can have on individuals and the healthcare system,” he added.

The agency based its approval on results from the randomized, double blind, placebo-controlled, multi-center study phase 3 study, which showed a significantly greater proportion of patients receiving Reblozyl achieving independence from red blood cell transfusions for at least eight weeks during the first 24 weeks of the trial compared with those receiving placebo.

In addition, a significantly greater proportion of patients also achieved at least 12 weeks of independence from transfusions within the first 24 and 48 weeks of the study.

Of note for patients, Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia, according to the release.

"Today's approval of Reblozyl is an important milestone for a majority of patients with myelodysplastic syndromes who have limited treatment options to address anemia associated with their disease. It also demonstrates our continued commitment to develop innovative products that improve the lives of patients living with serious diseases," Dr. Diane McDowell, vice president of hematology global medical affairs at Bristol Myers Squibb, said in the release. "We are looking forward to making Reblozyl immediately available for this patient population."


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