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

The FDA has approved luspatercept-aamt (Reblozyl, Bristol Myers Squibb) 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 (MDS) with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. It is the first and only erythroid maturation agent for this condition, according to the company.

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.

The approval is based on results from the phase 3 MEDALIST trial and marks the second indication for luspatercept-aamt, which received its first approval in November 2019 for the treatment of anemia in adults with beta thalassemia who require regular RBC transfusions.

A greater proportion of patients receiving luspatercept-aamt in the trial achieved independence from RBC transfusions for at least 8 weeks during the first 24 weeks of the trial compared with those receiving placebo, according to a press release. Additionally, a greater proportion of patients receiving luspatercept-aamt versus placebo achieved at least 12 weeks of independence from transfusions within the first 24 and 48 weeks of the study.

The major adverse reactions reported from the trial included fatigue, musculoskeletal pain, dizziness, diarrhea, dyspnea, nausea, hypersensitivity reactions, headache, and upper respiratory tract infection.

“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, MD, professor and chief of Section of Myelodysplastic Syndromes, Department of Leukemia, University of Texas MD Anderson Cancer Center, in a press release. “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 health care system.”

REFERENCE

U.S. Food and Drug Administration (FDA) Approves Reblozyl® (luspatercept-aamt), the First and Only Erythroid Maturation Agent, to Treat Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS). BusinessWire. https://www.businesswire.com/news/home/20200403005530/en/U.S.-Food-Drug-Administration-FDA-Approves-Reblozyl%C2%AE. Published April 3, 2020. Accessed April 6, 2020.