FDA Approves Luspatercept as First-Line Treatment of Anemia in Adults with Lower-Risk MDS

The Food and Drug Administration approved luspatercept-aamt (Reblozyl, Bristol Myers Squibb) on Monday as a first-line treatment for anemia adult patients with low- to intermediate risk myelodysplastic syndromes (MDS) without previous erythropoiesis stimulating agent (ESA) use who may require regular red blood cell transfusions.

The approval is based on interim results from the Phase 3 COMMANDS trial, in which luspatercept demonstrated superior efficacy of concurrent RBC transfusion independence (RBC-TI) and hemoglobin (Hb) increase compared to epoetin alfa, an ESA, regardless of ring sideroblast status.

In the trial, 58.5% of patients treated with luspatercept achieved the primary endpoint of RBC-TI of at least 12 weeks with a mean Hb increase of at least 1.5 g/dL within the first 24 weeks, compared with 31.2% of patients treated with epoetin alfa. The most common adverse reactions were diarrhea, fatigue, hypertension, peripheral edema, nausea and dyspnea.