CLINICAL NEWS | FDA Approves Luspatercept for First-line Treatment of Anemia in Lower-Risk MDS

The Food and Drug Administration (FDA) has approved Reblozyl® (luspatercept-aamt) as first-line treatment of anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions and are erythropoiesis stimulating agent (ESA)-naïve.

The approval was based on interim results from the pivotal phase 3 COMMANDS trial ( Identifier: NCT03682536) that evaluated the efficacy and safety of luspatercept in 356 patients with anemia due to international prognostic scoring system (IPSS-R) very low-, low-, or intermediate-risk MDS or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN RS-T) in ESA-naïve patients who require regular RBC transfusions.

Study participants were randomly assigned 1:1 to receive luspatercept or epoetin alfa for 24 weeks. All participants received best supportive care including RBC transfusions as needed. The primary endpoint was RBC transfusion independence (RBC-TI) for 12 weeks with a mean hemoglobin (Hgb) increase of at least 1.5g/dL.

Findings showed that 58.5% of patients treated with luspatercept achieved RBC-TI for at least 12 weeks with a mean Hgb increase of at least 1.5g/dL within the first 24 weeks vs 31.2% of patients treated with epoetin alfa (common rate difference, 26.6 [95% CI, 15.8-37.4]; <.0001).

Additionally, a significantly greater proportion of patients in the luspatercept treatment arm met key secondary endpoints within the first 24 weeks of treatment vs the epoetin-alfa arm, including hematologic improvement-erythroid increase of at least 8 weeks (74.1% vs 51.3%, respectively; <.0001); RBC-TI of at least 24 weeks (47.6% vs 29.2%, respectively; =.0012); and RBC-TI of at least 12 weeks (66.7% vs 46.1%, respectively; =.0003).

Reblozyl is also approved to treat anaemia in adults with beta-thalassaemia who require RBC transfusions; and for the treatment of anemia failing an ESA and requiring 2 or more RBC units over 8 weeks in adults with very low- to intermediate-risk MDS-RS or with MDS/MPN-RS-T.

Source: BMS Press Release

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