Bristol Myers Squibb gets USFDA priority review for expanded Reblozyl use
Reblozyl, a first-in-class therapeutic option, promotes late-stage red blood cell maturation in animal models.
Princeton: Bristol Myers Squibb today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) and the European Medicines Agency (EMA) has validated the Type II Variation Application for Reblozyl (luspatercept-aamt), a first-in-class treatment option, to expand its current indication to include treatment of anemia without previous use of erythropoiesis-stimulating agents (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require red blood cell (RBC) transfusions.
In the U.S., the FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 28, 2023. Priority Review designation underscores the high unmet need and value that Reblozyl could bring to this patient population. In Europe, the EMA’s validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
“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, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “Results from the COMMANDS study showed Reblozyl significantly improved transfusion independence and elevated hemoglobin compared to ESA therapy epoetin alfa. Reblozyl is an important option available for the treatment of anemia in patients with transfusion-dependent, lower-risk MDS who have experienced ESA failure, and we look forward to working with the FDA and EMA to expand its potential use as a first-line therapy in eligible patients.”
The submissions are based on results from the COMMANDS study, a Phase 3, open-label, randomized trial evaluating Reblozyl versus epoetin alfa, an ESA, for the treatment of anemia in adult patients with very low-, low- or intermediate-risk MDS who require RBC transfusions and are ESA-naïve. Results demonstrated a highly statistically significant and clinically meaningful improvement in red blood cell transfusion independence (RBC-TI) for ≥12 weeks with concurrent hemoglobin (Hb) increase (≥1.5 g/dL) 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 Reblozyl for the treatment of anemia in adult patients with low-risk MDS who require regular RBC transfusions, observed in previous clinical trials as well as in the post-marketing setting.
"Full results from the COMMANDS study will be presented at upcoming medical meetings," the company stated. Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.
