Sierpień 2021: The Food and Drug Administration approved idecabtagene vicleucel (Abecma, Bristol Myers Squibb) 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. This is the first cell-based gene therapy for multiple myeloma that has been approved by the FDA.
Idecabtagene vicleucel is a genetically engineered autologous chimeric antigen receptor (CAR) T-cell treatment that targets the B-cell maturation antigen (BCMA). Each dose is tailored to the patient’s own T-cells, which are harvested, genetically modified, and then reintroduced into the patient.
In a multicenter research, 127 patients with relapsed and refractory szpiczak mnogi who had undergone at least three prior lines of antimyeloma therapy were evaluated for safety and efficacy; 88 percent had received four or more prior lines of therapies. The efficacy of idecabtagene vicleucel at doses ranging from 300 to 460 x 106 CAR-positive T cells was studied in 100 individuals. The overall response rate (ORR), complete response (CR) rate, and duration of response (DOR) were calculated using the International Myeloma Working Group Uniform Response Criteria for Multiple Myeloma by an independent response committee.
ORR wyniósł 72 procent (95 procent CI: 62 procent, 81 procent), przy współczynniku CR 28 procent (95 procent CI 19 procent, 38 procent). W sumie 65 procent pacjentów, którzy osiągnęli CR, przebywało w nim przez co najmniej rok.
Ostrzeżenie w pudełku dla zespół uwalniania cytokin (CRS), neurologic toxicities, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, and persistent cytopenias is included on the idecabtagene vicleucel label. CRS, infections, exhaustion, musculoskeletal pain, and hypogammaglobulinemia are the most prevalent side effects of idecabtagene vicleucel.
Idecabtagene vicleucel has a risk evaluation and mitigation plan that requires healthcare facilities dispensing the medicine to be specially certified in recognising and managing CRS and nervous system toxicities. The FDA is ordering the company to conduct a post-marketing observational study involving patients treated with idecabtagene vicleucel in order to assess long-term safety.
Sugerowany zakres dawek dla idekabtagenu vicleucel to 300 do 460 106 limfocytów T CAR-dodatnich.
Odniesienie : https://www.fda.gov/
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