FDA approves luspatercept-aamt for anemia in adults with MDS

Share on facebook
Share on twitter
Share on linkedin
Share on email
Share on print

FDA has approved luspatercept-aamt (Reblozyl, sponsored by Celgene Corp) for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.

Efficacy was demonstrated in the MEDALIST trial (NCT02631070), a randomized, multi-center, double-blind, placebo-controlled trial in 229 patients with IPSS-R very low, low, or intermediate-risk myelodysplastic syndromes who had ring sideroblasts and required RBC transfusions (2 or more RBC units over 8 weeks). Patients were randomized 2:1 to luspatercept-aamt or placebo. All patients received best supportive care, which included RBC transfusions.

The main efficacy endpoint in MDS-RS and MDS-RS-T was the proportion of patients who were RBC-transfusion independent, defined as the absence of any RBC transfusion during any consecutive 8-week period between Weeks 1 and 24.

Of the 153 patients who received luspatercept-aamt, 58 (37.9%, 95% CI: 30.2, 46.1) were RBC-TI for at least 8 weeks, compared to 10 patients (13.2%, 95% CI: 6.5, 22.9) who received placebo (treatment difference 24.6% (95% CI: 14.5, 34.6; p<0.0001.)

Table of Contents

YOU MAY BE INTERESTED IN

The day before health economist Jay Bhattacharya stepped into his new role as NIH director, he sent a document to his employees outlining his top five priorities for the department, which included “reproducibility” and “transparency,” two themes he discussed at his confirmation hearings (The Cancer Letter, March 7, 2025).

Never miss an issue!

Get alerts for our award-winning coverage in your inbox.

Login