ADVERTISEMENT
FDA Approves Drug to Treat Anemia in Patients With MDS
On April 3, 2020, the FDA approved luspatercept-aamt (Reblozyl; Celgene) for the treatment of anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts or MDS/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. Specifically, the drug was approved for use in patients with anemia that didn’t respond to erythropoiesis stimulating agents and required ≥2 red blood cell (RBC) units over 8 weeks.
This approval was based on data from the double-blind, placebo-controlled MEDALIST clinical trial. A total of 229 patients with very low, low, or intermediate-risk MDS and ring sideroblasts according to the Revised International Prognostic Scoring System who required RBC transfusions were included in the study.
The main efficacy end point was the proportion of patients who were RBC-transfusion independent.
The trial randomized patients in a 2:1 ratio to receive luspatercept-aamt (n = 153) or placebo (n = 76). Of the 153 patients who received luspatercept-aamt, 58 were RBC-transfusion independent for at least 8 weeks compared with 10 patients who received placebo.
The most common (>10%) adverse events were fatigue, headache, musculoskeletal pain, arthralgia, dizziness/vertigo, nausea, diarrhea, cough, abdominal pain, dyspnea, and hypersensitivity.—Janelle Bradley
Source: US Food and Drug Administration. FDA approves luspatercept-aamt for anemia in adults with MDS. Updated April 6, 2020. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-luspatercept-aamt-anemia-adults-mds. Accessed April 6, 2020.
