The Food and Drug Administration (FDA) has approved Reblozyl® (luspatercept–aamt; Bristol-Myers Squibb and Acceleron) for the treatment of anemia in adults with lower-risk myelodysplastic syndromes.

Specifically, Reblozyl is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS), or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). 

The approval was based on data from the pivotal phase 3 MEDALIST trial that evaluated the efficacy and safety of Reblozyl in 229 adult patients with International Prognostic Scoring System-Revised (IPSS-R) very low-, low-, or intermediate-risk MDS-RS who require 2 or more RBC units over 8 weeks. Patients were randomized 2:1 to Reblozyl (n=153) or placebo (n=76). The primary end point was RBC transfusion independence (RBC-TI) of at least 8 weeks, over 24 weeks, defined as the proportion of patients who are RBC transfusion free over any consecutive 56-day period.

Results showed that Reblozyl met the primary end point with 37.9% of patients (n=58; 95% CI, 30.2-46.1) achieving RBC-TI compared with 13.2% of patients (n=10; 95% CI, 6.5-22.9) in the placebo arm (P <.0001). Additionally, a significantly greater proportion of patients treated with Reblozyl demonstrated at least 12 weeks of RBC-TI within the first 24 and 48 weeks with a common risk difference of 20% (P <.0002) and 21.4% (P <.0003), respectively, vs placebo.

With regard to safety, the most common adverse reactions observed were fatigue, musculoskeletal pain, dizziness, diarrhea, dyspnea, nausea, hypersensitivity reactions, headache, and upper respiratory tract infection. Grade 3 or 4 treatment-emergent adverse events were reported in 42.5% of Reblozyl-treated patients and 44.7% of placebo-treated patients.

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Reblozyl is an erythroid maturation agent that promotes late-stage red blood cell (RBC) maturation to help reduce RBC transfusion burden. In November 2019, Reblozyl received FDA approval for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions. 

“Today’s approval of Reblozyl is an important milestone for a majority of patients with myelodysplastic syndromes who have limited treatment options to address anemia associated with their disease,” said Diane McDowell, MD, vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. “We are looking forward to making Reblozyl immediately available for this patient population.”

Reblozyl is supplied as 25mg and 75mg lyophilized powder in single-dose vials for reconstitution and subcutaneous administration.

For more information visit bms.com and acceleronpharma.com.