Srpna 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 je geneticky upravená autologní léčba T-buněk autologního chimérního antigenního receptoru (CAR), která se zaměřuje na antigen zrání B-buněk (BCMA). Každá dávka je přizpůsobena pacientovým vlastním T-buňkám, které jsou sklizeny, geneticky modifikovány a poté znovu zavedeny do pacienta.
In a multicenter research, 127 patients with relapsed and refractory mnohočetný myelom 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 byla 72 procent (95 procent CI: 62 procent, 81 procent), s 28 procentní mírou CR (95 procent CI 19 procent, 38 procent). Celkem 65 procent pacientů, kteří dosáhli CR, v něm pobývali alespoň rok.
Upozornění v krabici pro syndrom uvolnění cytokinů (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.
300 až 460 106 CAR pozitivních T buněk je doporučené rozmezí dávek pro idecabtagene vicleucel.
Reference: https://www.fda.gov/
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