Reblozyl Approved to Treat Anemia in ESA-Naive, Lower-Risk MDS

The US Food and Drug Administration (FDA) has approved Reblozyl (luspatercept-aamt) to treat anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions and have not received prior treatment with erythropoiesis-stimulating agents (ESAs).

The approval was based on interim results from the phase 3 COMMANDS trial, which included 356 patients with ESA-naïve, transfusion-dependent, lower-risk MDS. The patients were randomly assigned to receive luspatercept (n=178) or epoetin alfa (n=178) for 24 weeks. All patients 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 1.5 g/dL. The percentage of patients who achieved the primary endpoint was 58.5% in the luspatercept arm and 31.2% in the epoetin alfa arm (P <.0001).

Patients in the luspatercept arm were also more likely than those in the epoetin-alfa arm to experience hematologic improvement-erythroid increase of at least 8 weeks (74.1% and 51.3%, respectively; P <.0001), RBC-TI of at least 24 weeks (47.6% and 29.2%, respectively; P =.0012), and RBC-TI of at least 12 weeks (66.7% and 46.1%, respectively; P =.0003). 

The most common adverse reactions observed with luspatercept were diarrhea, fatigue, hypertension, peripheral edema, nausea, and dyspnea.

Reblozyl is also approved for the treatment of anemia in adults with beta thalassemia who require RBC transfusions and for the treatment of anemia in adults with very low- to intermediate-risk MDS with ring sideroblasts or MDS/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis who require 2 or more RBC units over 8 weeks and experienced prior ESA failure.

References

1. US FDA approves Bristol Myers Squibb’s Reblozyl^® (luspatercept-aamt) as first-line treatment of anemia in adults with lower-risk myelodysplastic syndromes (MDS) who may require transfusions. News release. Bristol Myers Squibb. Accessed August 29, 2023. https://news.bms.com/news/details/2023/U.S.-FDA-Approves-Bristol-Myers-Squibbs-Reblozyl-luspatercept-aamt-as-First-Line-Treatment-of-Anemia-in-Adults-with-Lower-Risk-Myelodysplastic-Syndromes-MDS-Who-May-Require-Transfusions/default.aspx.

2. Package insert. Bristol Myers Squibb; 2023. Accessed August 29, 2023. https://packageinserts.bms.com/pi/pi_reblozyl.pdf

This article originally appeared on MPR