April 06, 2020
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FDA approves Reblozyl to treat anemia among adults with lower-risk myelodysplastic syndrome

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The FDA approved luspatercept-aamt to treat anemia among certain adults with lower-risk myelodysplastic syndrome.

The approval is the first in more than a decade for patients with myelodysplastic syndrome, according to a press release from the agent’s manufacturer.

It authorizes use of luspatercept-aamt (Reblozyl; Bristol-Myers Squibb, Acceleron) by adults with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis who have anemia that failed an erythropoiesis stimulating agent and requires two or more red blood cell units over 8 weeks.

Luspatercept-aamt, a erythroid maturation agent, is not indicated as a substitute for red blood cell transfusions for 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 syndrome who have ring sideroblasts,” Guillermo Garcia-Manero, MD, professor and chief of the section of myelodysplastic syndrome in the department of leukemia at The University of Texas MD Anderson Cancer Center, said in the press release.

“Anemia is a serious consequence of myelodysplastic syndrome, 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,” Garcia-Manero added. “In our current environment, we are reminded of the significant burden frequent blood transfusions can have on individuals and the health care system.”

The FDA previously approved luspatercept-aamt for treatment of anemia among adults with beta thalassemia who require red blood cell transfusions.

“[This] approval of Reblozyl is an important milestone for a majority of patients with myelodysplastic syndrome who have limited treatment options to address anemia associated with their disease,” Diane McDowell, MD, vice president of hematology global medical affairs with Bristol-Myers Squibb, said in the release.

The FDA based the approval on results of the randomized phase 3 MEDALIST study, which evaluated the efficacy and safety of luspatercept-aamt for patients with very low-, low- and intermediate-risk myelodysplastic syndrome who had ring sideroblasts but did not have deletion 5q.

All patients were red blood cell transfusion-dependent and either were intolerant of or refractory to or had not received prior erythropoiesis-stimulating agent therapy and had received no prior treatment with disease-modifying agents.

A higher percentage of patients assigned luspatercept-aamt than placebo achieved independence from red blood cell transfusions for at least 8 weeks during the first 24 weeks of the trial, the study’s primary endpoint.

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In addition, a significantly higher percentage of patients assigned luspatercept-aamt achieved at least 12 weeks of independence from transfusion within the first 24 weeks and first 48 weeks of the study.

A comparable percentage of patients assigned luspatercept-aamt and placebo experienced grade 3 or grade 4 treatment-emergent adverse events (42.5% vs. 44.7%). The most common all-grade adverse events included fatigue, musculoskeletal pain, dizziness, diarrhea, dyspnea, nausea, hypersensitivity reactions, headache and upper respiratory tract infection.