FDA approved Reblozyl (luspatercept-aamt) for Anemia in Beta Thalassemia and MDSFDA approved Reblozyl for Anemia in Three Adult Blood Disorder Groups

Reblozyl is an erythroid maturation agent. The label does not provide further mechanistic detail.

Reblozyl is indicated for anemia in adult patients with beta thalassemia who require regular RBC transfusions. It is also indicated for anemia without previous erythropoiesis stimulating agent use (ESA-naive) in adults with very low- to intermediate-risk MDS who may require regular RBC transfusions. It is further indicated for anemia failing an erythropoiesis stimulating agent and requiring 2 or more RBC units over 8 weeks in adults with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with MDS/MPN-RS-T. Limitation of use: Reblozyl is not indicated as a substitute for RBC transfusions in patients who require immediate correction of anemia.

For beta thalassemia, the starting dose is 1 mg/kg once every 3 weeks by subcutaneous injection. If there is no reduction in RBC transfusion burden after at least 2 consecutive doses (6 weeks) at 1 mg/kg, increase to 1.25 mg/kg every 3 weeks. If there is no reduction in transfusion burden after 3 consecutive doses (9 weeks) at 1.25 mg/kg, discontinue. Do not exceed 1.25 mg/kg. For predose hemoglobin of 11.5 g/dL or greater in the absence of transfusion, interrupt and restart at the same dose when hemoglobin is 11 g/dL or less. For a hemoglobin rise greater than 2 g/dL within 3 weeks without transfusion, reduce dose by level (1.25 to 1 mg/kg, 1 to 0.8 mg/kg, 0.8 to 0.6 mg/kg) or discontinue at 0.6 mg/kg. For MDS, the starting dose is 1 mg/kg every 3 weeks subcutaneously. In ESA-naive MDS, increase to maintain hemoglobin within 10 to 12 g/dL, to a maximum of 1.75 mg/kg. In ESA-refractory or intolerant MDS, increase if the patient is not RBC transfusion-free, to a maximum of 1.75 mg/kg. Review hemoglobin before each administration. Grade 3 or 4 hypersensitivity reactions require discontinuation; other Grade 3 or 4 adverse reactions require interruption and restart at the next lower dose once resolved to Grade 1 or less.

Trial data not available in label.

Contraindications: Not reported in label. Warnings and precautions: Not reported in label. The label specifies dose interruption and discontinuation criteria for Grade 3 or 4 adverse reactions and hypersensitivity reactions.

Reblozyl offers a subcutaneous, every-3-week erythroid maturation approach for adults with transfusion-dependent beta thalassemia and for defined MDS populations, including ESA-naive patients and those failing an ESA with ring sideroblasts. It is not a substitute for RBC transfusions when immediate correction of anemia is required.