FDA Accepts BLA for Luspatercept-aamt as Treatment of Anemia in MDS

The US Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (BLA) for luspatercept-aamt to expand the current indication of the drug to include treatment of anemia without previous use of erythropoiesis-stimulating agents (ESAs) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require red blood cell (RBC) transfusions. The FDA has set a target action date of August 28, 2023.

The Type II Variation Application was accepted by the European Medicines Agency (EMA) as well. Both regulatory agencies based their decisions on results from the phase III COMMANDS study. In that trial, luspatercept-aamt demonstrated a statistically significant and clinically meaningful improvement compared with an ESA in red blood cell transfusion independence (RBC-TI) in the first-line treatment of adult patients with very low-, low-, or intermediate-risk MDS who require RBC transfusions.

Safety results in the trial were consistent with the safety profile of luspatercept-aamt for the treatment of anemia in adult patients with low-risk MDS who require regular RBC transfusions as observed in previous clinical trials.

The primary endpoint evaluated in the COMMANDS study is RBC-TI for 12 weeks with a mean hemoglobin increase ≥1.5 g/dL. Key secondary endpoints include RBC-TI for 24 weeks, RBC-TI ≥12 weeks, and erythroid response of at least eight weeks during weeks 1-24 of the study.

Luspatercept-aamt, a first-in-class therapeutic option, promotes late-stage RBC maturation in animal models.

Source: Bristol Myers Squibb, May 2023