Positive CHMP opinion for CAR T Cell therapy Abecma (idecabtagene vicleucel)in earlier lines of therapy for triple-class exposed relapsed and refractory multiple myeloma.- BMS

The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.

“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 III, 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.

The FDA has delayed a decision, however, and intends to seek input from an advisory committee to review patient survival data.