The U.S. Food and Drug Administration (FDA) has approved luspatercept (Reblozyl) as first in line therapy for anemia in adult patients with myelodysplastic syndromes (MDS). More specifically, the drug is approved to treat anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions. This approval expands on an earlier indication that limited the drug’s use for those patients who had previous had ESA treatment that was unsuccessful.
MDS, as defined by the NIH, are a group of blood disorders marrow in which the bone marrow makes too many white blood cells. The three main types of MDS include chronic myelomonocytic leukemia (CMML); juvenile myelomonocytic leukemia (JMML); and atypical chronic myelogenous leukemia (aCML). People with MDS who develop anemia often require red blood cell transfusions.
The approval to expand the indication of luspatercept was largely based on interim results from the pivotal Phase 3 COMMANDS trial showing 58.5% (n=86) of patients treated with luspatercept achieved RBC transfusion independence of at least 12 weeks compared to 31.2% (n=48) of patients treated with epoetin alfa. The most common adverse reactions were diarrhea, fatigue, hypertension, peripheral edema, nausea, and dyspnea. These results were recently published in The Lancet.
To stay up-to-date on the latest FDA approvals and PDUFA dates, visit checkrare.com/2023-orphan-drugs-pdufa-dates-and-fda-approvals/
Reference
Platzbecker U, Della Porta MG, Santini V, et al. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. The Lancet. 2023 Jul 29; 402: 373-385.
