Elokuu 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 on geneettisesti muokattu autologinen kimeerisen antigeenireseptorin (CAR) T-solujen hoito, joka kohdistuu B-solujen kypsymisantigeeniin (BCMA). Jokainen annos räätälöidään potilaan omien T-solujen mukaan, jotka kerätään, muunnetaan geneettisesti ja viedään sitten takaisin potilaaseen.
In a multicenter research, 127 patients with relapsed and refractory multippeli myelooma 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 oli 72 prosenttia (95 prosenttia luottamusväli: 62 prosenttia, 81 prosenttia) ja 28 prosentin CR -korko (95 prosenttia luottamusväli 19 prosenttia, 38 prosenttia). Yhteensä 65 prosenttia potilaista, jotka saavuttivat CR: n, pysyivät siinä vähintään vuoden ajan.
Laatikollinen varoitus sytokiinien vapautumisoireyhtymä (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.
300-460 CAR-positiivista T-solua on ehdotettu annosväli idecabtagene vicleucelille.
Viite: https://www.fda.gov/
Tarkista tiedot tätä:
