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FDA grants approval to luspatercept-aamt for the treatment of anemia in adults with myelodysplastic syndromes

By Paola Frisone

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Apr 7, 2020


On April 3, 2020, the United States Food and Drug Administration granted approval to luspatercept-aamt (hereafter designated luspatercept) for the treatment of anemia in adults with very low-, low-, or intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who require regular red blood cell (RBC) transfusions and failed an erythropoiesis stimulating agent (ESA).1

Luspatercept is an erythroid maturation agent promoting late-stage red blood cell maturation. Its approval is based on the phase III MEDALIST trial (NCT02631070) in which patients, with MDS-RS or MDS/MPN-RS-T who were RBC transfusion dependent and who failed prior ESA therapy or were ESA naïve, were randomized to receive luspatercept or placebo.1 A higher proportion of patients treated with luspatercept (38%) showed RBC transfusion independence for at least 8 weeks within the first 24 weeks, compared to the placebo group (13%; p < 0.001). Additionally, a higher percentage of patients treated with luspatercept showed at least 12 weeks of independence in the first 24 weeks (28% vs 8% in luspatercept vs placebo group, respectively; p < 0.001) and 48 weeks (33% vs 12% in luspatercept vs placebo group, respectively; p < 0.001) of the trial compared to the placebo group.2

Treatment-emergent adverse events of Grade 3 or 4 were reported in 42.5% of patients who received luspatercept vs 44.7% of patients who received placebo.1 The most common (occurring in ≥ 10% of patients) all-grade adverse events included fatigue, diarrhea, asthenia, nausea, dizziness, and back pain.2

This approval marks the second indication for luspatercept, which was already approved in the United States for the treatment of patients with beta thalassemia.

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