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FDA Approves Luspatercept as First-Line Anemia Treatment in Patients With Lower-Risk MDS


The FDA has approved an expanded indication for luspatercept (Reblozl) for the first-line treatment of anemia in patients with low-risk MDS who may require blood transfusions.

The FDA has approved luspatercept (Reblozl) for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (MDS) who may need red blood cell (RBC) transfusions and are naïve to erythropoiesis-stimulating agents (ESAs).1

The approval was supported by data from the COMMANDS study, a global phase 3 trial in which 58.5% (n = 86) of patients with lower-risk MDS at risk of anemia who were treated with first-line luspatercept were able to forego transfusions for 12 weeks compared with 31.2% (n = 48) of patients who received epoetin alfa, an ESA. Findings from the trial were presented at the 2023 American Society of Clinical Oncology Annual Meeting.2

“For patients with lower-risk MDS, current standard therapies, including ESAs, have provided limited benefit in controlling anemia with only 1 in 3 patients responding for a duration of 6-18 months,” Guillermo Garcia-Manero, MD, lead investigator and chief of the Section of Myelodysplastic Syndromes at the University of Texas MD Anderson Cancer Center, said in a statement. “Results from the COMMANDS study showed nearly twice as many patients treated with Reblozyl achieved transfusion independence of at least 12 weeks and concurrent hemoglobin increase compared to epoetin alfa. Today’s approval represents an important advancement for patients with lower-risk MDS.”1

Compared with the current first-line treatment standard of ESAs, luspatercept has shown the capability to decrease the necessary frequency of RBC transfusions and, subsequently, visits to the physician’s office. A major advantage to luspatercept is that injections are only administered once every 3 weeks, compared to weekly injections needed with ESAs.

In the COMMANDS trial, patients were randomized into 2 groups to assess responses to luspatercept and epoetin alfa. The primary end point was transfusion independence for at least 12 weeks, with a mean hemoglobin increase of at least 15 g/dL within the initial 24 weeks of treatment. Secondary end points included a hematologic improvement-erythroid (HI-E) response after at least 8 weeks and transfusion independence at 24 weeks (for at least 12 weeks).

The planned interim analysis included 301 patients who had not yet received ESAs. The luspatercept group showed significantly higher rates of transfusion independence at 12 weeks compared with the group receiving epoetin alfa (58.5% vs 31.2%; P < .0001).

Among the two groups, treatment-emergent adverse events (TEAEs) were reported in 92.1% of luspatercept patients and 85.2% of epoetin alfa, respectively. Additionally, treatment-related AEs were documented by 30.3% of patients receiving luspatercept and 17.6% receiving epoetin alfa.

The drug was previously approved by the FDA for the treatment of adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who have anemia failing an ESA and requiring 2 or more RBC units over 8 weeks.3


1. U.S. 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. August 28, 2023. 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. Caffrey, M. COMMANDS: Luspatercept boosts population with lower-risk myelodysplastic syndromes who are freed from blood transfusions. AJMC. Accessed August 28, 2023. https://www.ajmc.com/view/commands-luspatercept-boosts-population-with-lower-risk-myelodysplastic-syndromes-who-are-freed-from-blood-transfusions

3. FDA approves luspatercept-aamt for anemia in adults with MDS, News release. FDA. April 3, 2020. Accessed August 29, 2023. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-luspatercept-aamt-anemia-adults-mds

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