Bristol Myers Squibb announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization approval of Abecma® (idecabtagene vicleucel; ide-cel) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an anti-CD38 monoclonal antibody. The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation. 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.