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

Officials with the FDA have approved luspatercept-aamt (Reblozyl, Bristol Myers Squibb and Acceleron Pharma) for the treatment of anemia in adults with lower-risk myelodysplastic syndromes (MDS), making it the first and only erythroid maturation agent for patients with MDS, according to a press release.

With this approval, luspatercept-aamt is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adults with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Luspatercept-aamt was initially approved in November 2019 for the the treatment of anemia in adults with beta thalassemia who require regular RBC transfusions.

Luspatercept-aamt is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

The approval is based on data from the phase 3 MEDALIST trial, which evaluated the safety and efficacy of luspatercept-aamt in patients with IPSS-R-defined very low-, low-, and intermediate-risk non-del(5q) MDS-RS.

In the trial, a significantly greater proportion of patients receiving luspatercept-aamt achieved independence from RBC transfusions for at least 8 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 luspatercept-aamt versus placebo achieved at least 12 weeks of independence from transfusions within the first 24 and 48 weeks of the study.

The most common all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, dyspnea, nausea, hypersensitivity reactons, 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,” Guillermo Garcia-Manero, MD, professor and chief of Section of Myelodysplastic Syndromes, Department of Leukemia, University of Texas MD Anderson Cancer Center, said in a statement. “Anemia is a serious consequences 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.”

References:

1. US 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) [news release]. Bristol Myers Squibb’s website. https://news.bms.com/press-release/corporatefinancial-news/us-food-and-drug-administration-fda-approves-reblozyl-luspater. Accessed April 3, 2020.