Αύγουστος 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.
Το Idecabtagene vicleucel είναι μια γενετικά τροποποιημένη θεραπεία αυτόλογου χιμαιρικού αντιγόνου υποδοχέα χιμαιρικού αντιγόνου (CAR) που στοχεύει στο αντιγόνο ωρίμανσης των Β-κυττάρων (BCMA). Κάθε δόση προσαρμόζεται στα Τ-κύτταρα του ίδιου του ασθενούς, τα οποία συλλέγονται, τροποποιούνται γενετικά και στη συνέχεια εισάγονται ξανά στον ασθενή.
In a multicenter research, 127 patients with relapsed and refractory πολλαπλό μυέλωμα 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.
Το ORR ήταν 72 % (95 % CI: 62 %, 81 %), με ποσοστό CR 28 % (95 % CI 19 %, 38 %). Συνολικά το 65 τοις εκατό των ασθενών που πέτυχαν CR έμειναν σε αυτό για τουλάχιστον ένα χρόνο.
Μια προειδοποίηση σε κουτί για σύνδρομο απελευθέρωσης κυτοκίνης (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 έως 460 106 C-θετικά Τ κύτταρα είναι το προτεινόμενο εύρος δόσεων για το idecabtagene vicleucel.
Αναφορά: https://www.fda.gov/
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