Recommendation for approval based on Phase 3 KarMMa-3 study in which Abecma demonstrated superiority over standard regimens, significantly improved progression-free survival and a well-established safety profile with mostly low-grade occurrences of cytokine release syndrome and neurotoxicity

This is the first positive CHMP opinion in earlier lines of therapy for a chimeric antigen receptor (CAR) T cell therapy in the treatment of relapsed and refractory multiple myeloma

Approval by the European Commission would expand Abecma’s indication, making it the first CAR T cell therapy available in the European Union (EU) for patients with triple-class exposed relapsed and refractory multiple myeloma earlier in the treatment journey

“This positive CHMP opinion represents an important step toward bringing our potentially transformative first-in-class anti-BCMA CAR T cell therapy, Abecma, to more patients earlier in the multiple myeloma treatment paradigm to improve outcomes,” said Anne Kerber, M.D., senior vice president and head, Late Clinical Development, Hematology, Oncology, Cell Therapy (HOCT), Bristol Myers Squibb. “We look forward to working with the European Commission with the shared goal of delivering innovative treatment options to more patients with continued unmet need.”

The CHMP adopted a positive opinion based on the final progression-free survival (PFS) analysis from the pivotal, Phase 3, open-label, global, randomized, controlled KarMMa-3 study evaluating Abecma compared with standard combination regimens in adults with relapsed and refractory multiple myeloma after two to four prior lines of therapy, including an IMiD, a PI, and an anti-CD38 monoclonal antibody, which are the three main classes of therapy (triple-class exposed) in multiple myeloma, and who were refractory to their last regimen. Results recently presented at the American Society of Hematology (ASH) Annual Meeting in December 2023 showed, at a median follow-up of 30.9 months (range: 12.7-47.8), Abecma significantly improved PFS compared with standard regimens, with a median PFS of 13.8 months vs. 4.4 months (HR:0.49; 95% CI: 0.38-0.63), representing a 51% reduction in the risk of disease progression or death with Abecma.

Results for the key secondary endpoint of overall response rate showed the majority of patients (71%; (95% CI: 66-77) treated with Abecma achieved a response, with 44% (95% CI: 38-50) achieving a complete response or stringent complete response. In comparison, less than half of patients (41%; 95% CI: 34-51) who received standard regimens achieved a response, with 5% (95% CI: 2-9) experiencing a complete response or stringent complete response.

Treatment with Abecma exhibited a well-established safety profile, with mostly low-grade and transient occurrences of cytokine release syndrome (CRS) and neurotoxicity. In patients treated with Abecma, 88% experienced any grade CRS, with Grade 3/4 events occurring in 4% of patients. Two patients (1%) experienced a Grade 5 CRS event. Any grade neurotoxicity occurred in 15% of patients, with Grade 3/4 neurotoxicity occurring in 3% of patients, and no Grade 5 events reported.

In the EU, the EC delivers its final decision approximately two months following receipt of the CHMP opinion. The decision will be applicable to all EU member states and Iceland, Norway and Liechtenstein.* Please see the Important Safety Information section below, including Boxed WARNINGS for Abecma regarding cytokine release syndrome, neurologic toxicities, Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome and Prolonged Cytopenia. A supplemental Biologics License Application for Abecma in earlier lines of therapy for triple-class exposed relapsed and refractory multiple myeloma is currently under review by the U.S. Food and Drug Administration (FDA), and the Oncologic Drugs Advisory Committee of the FDA will convene a meeting to review data from KarMMa-3 supporting the application. Abecma is also approved in Japan for adult patients with triple-class exposed relapsed or refractory multiple myeloma after two prior lines of therapy, and in the EU, Switzerland, the United Kingdom, Canada, and Israel for adult patients with triple-class exposed relapsed and/or refractory multiple myeloma after three to four or more prior lines of therapy.

Bristol Myers Squibb thanks the patients and investigators involved in the KarMMa-3 study.

*Centralized Marketing Authorization does not include approval in Great Britain (England, Scotland and Wales).