FDA approves Abecma the first Anti-BCMA CAR T cell therapy for relapsed or refractory multiple myeloma.- BMS + bluebird bio

Bristol Myers Squibb and bluebird bio, Inc. announced that the FDA has approved Abecma (idecabtagene vicleucel; ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is a personalized immune cell therapy approved as a one-time infusion with a recommended dose range of 300 to 460 x 106 CAR-positive T cells. As an anti-BCMA CAR T cell therapy, Abecma recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells. The FDA approval of Abecma is based on data from the pivotal Phase II KarMMa trial of 127 patients with relapsed or refractory multiple myeloma who had received at least three prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The efficacy evaluable population consists of 100 patients who received Abecma within the dose range of 300 to 460 x 106 CAR-positive T cells. Of these patients, 88% received four or more prior lines of therapy and 85% were triple-class refractory. In the study, the overall response rate (ORR) for the efficacy evaluable population (n=100) was 72% (95% CI: 62-81), and 28% of patients achieved a stringent complete response (sCR; 95% CI: 19-38). Responses were rapid and durable, with a median time to response of 30 days (range: 15 to 88 days) and median duration of response of 11 months (95% CI: 10.3 – 11.4) for all responders and 19 months (95% CI: 11.4 – NE) for those who achieved sCR. Of the 28 patients who achieved sCR, an estimated 65% (95% CI: 42% - 81%) had remission lasting at least 12 months. In patients treated with Abecma in the KarMMa study, the safety profile was well-established with mostly low-grade occurrence of cytokine release syndrome (CRS) and neurotoxicity (NT), and predictable early onset and resolution. CRS of any grade occurred in 85% (108/127) of patients using the Lee grading system. Grade >3 CRS occurred in 9% (12/127) of patients, with Grade 5 CRS reported in one patient (0.8%). Abecma is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the ABECMA REMS.