FDA to evaluate luspatercept-aamt (Reblozyl) for the treatment of anemia in adults with non–transfusion dependent β-thalassemia.
The FDA has granted priority review to a supplemental biologics license application (sBLA) for luspatercept-aamt (Reblozyl) for the treatment of anemia in adults with non–transfusion dependent (NTD) β-thalassemia. The drug was also validated by the European Medicines Agency for the Type II variation in the same indication.1
“Patients with non-transfusion dependent beta thalassemia may not require lifelong blood transfusions for survival, but their need for effective treatment options is significant as they face a range of clinical complications due to chronic anemia and iron overload,” said Noah Berkowitz, MD, PhD, senior vice president, Hematology Development, Bristol Myers Squibb, in a press release. “Reblozyl is an important therapy approved for anemia associated with beta thalassemia and lower-risk myelodysplastic syndromes in multiple countries, including the United States and within the European Union. Along with our partners at Merck, we are committed to continuing to advance our clinical program for Reblozyl and look forward to working with the FDA during its review of our application for this underserved patient population.”
The phase 2, double-blind, randomized, placebo-controlled, multicenter BEYOND study evaluated the efficacy and safety of luspatercept-aamt compared with placebo in adults with NTD beta thalassemia. The study was divided into a screening period, double-blind treatment period and post-treatment follow-up period. The researchers randomized 145 patients at a 2:1 ratio of Reblozyl versus placebo.
The primary endpoint was the proportion of patients who have an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from week 13 to week 24 of treatment in the absence of transfusions. Key secondary endpoints were mean change in non-transfusion dependent beta thalassemia-patient-reported outcome tiredness and weakness domain score, and baseline hemoglobin.
In the trial, luspatercept achieved a 77.1% mean hemoglobin (Hb) increase of 1.0 g/dL or higher from baseline over a continuous 12-week interval from weeks 13 to 24 in the absence of red blood cell (RBC) transfusions vs 0% with placebo in patients with NTD β-thalassemia, meeting the primary end point of the trial.
Luspatercept-aamt, the first and only erythroid maturation agent, was found to promote late-stage, red blood cell maturation in animal models. It is currently FDA-approved for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions; and anemia failing an erythropoiesis stimulating agent requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.
Luspatercept-aamt is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.
The FDA is expected to decide on the sBLA for the current indication by March 27, 2022, under the Prescription Drug User Fee Act.
1. US Food and Drug Administration accepts for priority review supplemental biologics license application for Reblozyl (luspatercept-aamt) in adults with non-transfusion dependent (NTD) beta thalassemia. News release. Bristol Myers Squibb; December 3, 2021. Accessed December 3, 2021. https://bit.ly/3rCHQsy