August 2021: Food and Drug Administration godkendte idecabtagene vicleucel (Abecma, Bristol Myers Squibb) til behandling af voksne patienter med recidiverende eller refraktær myelomatose efter fire eller flere tidligere behandlingslinjer, inklusive et immunmodulerende middel, en proteasomhæmmer og en anti-CD38 monoklonalt antistof. Dette er den første cellebaserede genterapi for myelomatose, som er blevet godkendt af FDA.
Idecabtagene vicleucel er en gensplejset autolog kimær antigenreceptor (CAR) T-cellebehandling, der retter sig mod B-cellemodningsantigenet (BCMA). Hver dosis er skræddersyet til patientens egne T-celler, som høstes, genmodificeres og derefter genindføres i patienten.
In a multicenter research, 127 patients with relapsed and refractory multiple 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 var 72 procent (95 procent CI: 62 procent, 81 procent), med en CR-rate på 28 procent (95 procent CI 19 procent, 38 procent). I alt 65 procent af patienterne, der opnåede CR, blev i det i mindst et år.
En indrammet advarsel for cytokinfrigivelsessyndrom (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 til 460 106 CAR-positive T-celler er det foreslåede dosisområde for idecabtagene vicleucel.
Reference: https://www.fda.gov/
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