Luspatercept granted priority review by the FDA and type II variation application validated by the EMA for first-line treatment of anemia in lower-risk MDS

On May 1, 2023, the U.S. Food and Drug Administration (FDA) accepted a supplemental biologics license application and granted priority review to luspatercept-aamt, a first-in-class erythroid-maturation agent, for the treatment of anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have not previously been treated with erythropoiesis-stimulating agents (ESAs).¹ The European Medicines Agency (EMA) has also validated the type II variation application for luspatercept-aamt for the same indication.¹

Luspatercept-aamt has previously been approved by both the FDA and EMA for the treatment of anemia in patients with MDS with ring sideroblasts (MDS-RS) who are transfusion dependent and either do not respond to or are ineligible for ESA treatment. These approvals were based on results from the phase III MEDALIST trial (NCT02631070), previously covered on the MDS Hub. The latest submissions are based on results from the phase III, open-label, randomized COMMANDS trial (NCT03682536), investigating the efficacy and safety of luspatercept-aamt versus epoetin alfa, an ESA, for the treatment of patients with anemia with very low- to intermediate-risk MDS who require red blood cell (RBC) transfusions and have not previously received ESA treatment.¹ Results indicate that first-line treatment of this patient population with luspatercept-aamt improves RBC transfusion independence for ≥12 weeks, with a concurrent hemoglobin increase of ≥1.5 g/dL, compared with epoetin alfa.¹ The safety profile of luspatercept-aamt was consistent with previous studies.¹