August 2021: Food and Drug Administration godkjente idecabtagene vicleucel (Abecma, Bristol Myers Squibb) for behandling av voksne pasienter med residiverende eller refraktær multippelt myelom etter fire eller flere tidligere behandlingslinjer, inkludert et immunmodulerende middel, en proteasomhemmer og en anti-CD38 monoklonalt antistoff. Dette er den første cellebaserte genterapien for multippelt myelom som er godkjent av FDA.
Idecabtagene vicleucel er en genetisk konstruert autolog kimær antigenreseptor (CAR) T-cellebehandling som retter seg mot B-cellemodningsantigenet (BCMA). Hver dose er skreddersydd til pasientens egne T-celler, som høstes, genmodifiseres og deretter gjeninnføres i pasienten.
In a multicenter research, 127 patients with relapsed and refractory flere myelomer 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 prosent (95 prosent KI: 62 prosent, 81 prosent), med en 28 prosent CR -rate (95 prosent KI 19 prosent, 38 prosent). Totalt 65 prosent av pasientene som oppnådde CR ble i det i minst et år.
En eske advarsel for cytokinfrigjøringssyndrom (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 foreslåtte doseområdet for idecabtagene vicleucel.
Henvisning : https://www.fda.gov/
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