The US FDA accepted BMS’ supplemental Biologics License Application (sBLA) and granted Priority Review for Reblozyl, while the European Medicines Agency (EMA) has also validated the company’s Type II Variation Application for Reblozyl
Bristol Myers Squibb building signage. (Credit: Bristol-Myers Squibb Company)
Bristol Myers Squibb (BMS) has received the US Food and Drug Administration (FDA) Priority Review for its anaemia drug Reblozyl (luspatercept-aamt) to expand its current indication.
The expanded indication includes the treatment of anaemia in adults with very low-risk myelodysplastic syndromes (MDS), who are not previously treated with erythropoiesis-stimulating agents and may require red blood cell (RBC) transfusions.
The US agency has accepted the drugmaker’s supplemental Biologics License Application (sBLA) and assigned a Prescription Drug User Fee Act (PDUFA) goal date of 28 August 2023.
In addition, BMS announced that the European Medicines Agency (EMA) has validated its Type II Variation Application for Reblozyl, which marks the start of the review process by EMA.
Reblozyl is a recombinant fusion protein derived from human activin receptor type IIb (ActRIIb), previously approved to treat anaemia in beta-thalassemia and myelodysplastic syndromes.
BMS is developing and commercialising the drug under a collaboration agreement with Merck, signed after Merck acquired Acceleron Pharma in November 2021.
The company’s regulatory submissions are supported by results from the Phase 3 COMMANDS clinical trial, which evaluated Reblozyl compared to epoetin alfa, an ESA.
In the study results, the treatment using Reblozyl showed a highly statistically significant and clinically meaningful improvement in red blood cell transfusion independence (RBC-TI).
The drug showed safety results consistent with the safety profile of Reblozyl, observed in previous clinical trials along with the post-marketing setting.
Bristol Myers Squibb haematology development senior vice president Noah Berkowitz said: “Results from the COMMANDS study showed Reblozyl significantly improved transfusion independence and elevated haemoglobin compared to ESA therapy epoetin alfa.
“Reblozyl is an important option available for the treatment of anaemia 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.”
In a separate development, BMS has unveiled positive study results for its cancer drug Breyanzi (lisocabtagene maraleucel), from TRANSCEND FL, and TRANSCEND NHL 001 studies.
TRANSCEND FL is an open-label, global, multicentre, single-arm Phase 2 study evaluating Breyanzi in patients with relapsed or refractory follicular lymphoma (FL).
TRANSCEND NHL 001 is an open-label, multicentre, single-arm Phase 1 study evaluating Breyanzi in patients with B-cell non-Hodgkin lymphoma, including mantle cell lymphoma (MCL).
In both studies, which met the primary endpoint of overall response rate, Breyanzi showed statistically significant and clinically meaningful responses in relapsed or refractory FL and MCL.
The studies also met the key secondary endpoint of complete response rate, in both relapsed or refractory FL and MCL, with no new safety signals in either disease.
Bristol Myers Squibb senior vice president and cell therapy development head Anne Kerber said: “For people living with relapsed or refractory follicular lymphoma or mantle cell lymphoma, there are limited treatment options that provide deep and durable responses, especially for patients with high-risk disease.
“These continued unmet needs coupled with our deep understanding of lymphoma biology drive us to deliver transformative treatments for patients.
“We believe these data further confirm Breyanzi’s best-in-class and best-in-disease profile and underscore the significant progress we are making in bringing the promise of our differentiated CAR T cell therapy, Breyanzi, to more patients.”