Bristol-Myers Squibb (BMS) and bluebird bio announced that the Food and Drug Administration (FDA) has approved the marketing of Abecma (idecabtagene vicleucel), a chimeric antigen receptor T (CAR-T) cell immunotherapy targeting B cell mature antigen (BCMA), for the treatment of adult patients with relapsed/refractory multiple myeloma (R/R MM) who have received more than four previous treatments. This is the first FDA-approved CAR-T cell therapy that targets BCMA.
Multiple Myeloma (MM) is characterized by abnormal proliferation of bone marrow plasma cells and excessive production of monoclonal immunoglobulin. Abnormal plasma cells gather in the bone marrow and form tumors in many bone tissues in the body. These cells not only fail to perform their normal functions, but also produce antibodies that prevent the bone marrow from producing healthy blood cells. In addition, patients will also be accompanied by multiple osteolytic damages, hypercalcemia, anemia, kidney damage. Although a variety of treatments have been developed to treat multiple myeloma, there are still many patients who are resistant to all approved treatments. R/R MM patients who have been treated with immunomodulators, proteasome inhibitors and anti-CD38 antibodies have a poor prognosis. Therefore, it is very important to develop innovative therapies.
BCMA is a protein commonly expressed in cancer cells of multiple myeloma. It is an important potential target for this invasive hematological cancer. Abecma can recognize and bind to BCMA on the surface of multiple myeloma cells, which leads to the proliferation and differentiation of CAR-T cells, and then kills the cells expressing BCMA. Prior to FDA’s approval, Abecma has been granted the breakthrough therapy designation by FDA and has been recognized as the Priority Medicines (PRIME) by the European Drug Administration (EDA).
FDA’s approval of Abecma is based on data from the critical Phase II clinical trial KarMMa, which treated 127 patients with R/R MM who received at least three prior treatments, including immunomodulators, proteasome inhibitors and anti-CD38 antibodies. The population that could evaluate the efficacy was composed of 100 patients who received Abecma treatment. Of these patients, 88% had received four or more prior treatments, and 85% of patients developed resistance to three treatments.
In this study, the total remission rate of the evaluable population was 72% (95% CI:62%-81%), and 28% of patients achieved strict complete remission (sCR; 95% CI:19%-38%). The patient produced a rapid and sustained response with a median time of 30 days and a median duration of 11 months (95% CI:10.3-11.4). Of the 28 patients with strict complete remission, 65% (95% CI:42%-81%) sustained remission for at least 12 months.
“CAR-T cell therapy has shown revolutionary potential for the treatment of hematological malignancies, and together with our partner bluebird bio, we are honored to bring this CAR-T cell therapy to patients with recurrent/refractory multiple myeloma, providing them with the opportunity for lasting remission.” Dr. Samit Hirawat, Chief Medical Officer of BMS, said, “Bristol-Myers Squibb now has two FDA-approved CAR-T cell therapies targeting CD19 and BCMA. Abecma highlights our commitment to provide cell therapy for patients with invasive and advanced hematological cancer with limited treatment options. “