Luspatercept Gets Priority Review for First-Line Tx of Anemia in Lower-Risk MDS

The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for luspatercept-aamt as a first-line treatment of anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require red blood cell (RBC) transfusions.

Luspatercept-aamt is an erythroid maturation agent. It is currently approved under the brand name Reblozyl^® for the treatment of anemia failing an erythropoiesis stimulating agent (ESA) and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.

“Initial treatment options for very low- to intermediate-risk myelodysplastic syndromes, including erythropoiesis-stimulating agents, can alleviate anemia in some patients but others will either not respond or become resistant to therapy, and additional therapy options have remained urgently needed,” said Noah Berkowitz, MD, PhD, senior vice president, Hematology Development, Bristol Myers Squibb.

The sBLA includes data from the phase 3 COMMANDS study (ClinicalTrials.gov Identifier: NCT03682536), which compared the efficacy and safety of luspatercept vs epoetin alfa for the treatment of anemia due to very low-, low- or intermediate-risk MDS in patients who are RBC transfusion-dependent and ESA-naïve.

Findings showed that luspatercept was associated with a statistically significant and clinically meaningful improvement in RBC transfusion independence (≥12 weeks) with concurrent hemoglobin increase (≥1.5g/dL) (primary endpoint). Full results from the trial are expected at an upcoming medical meeting.

The FDA has assigned a Prescription Drug User Fee Act goal date of August 28, 2023 to finalize review of the application.