FDA approval summary: Lifileucel for unresectable or metastatic melanoma previously treated with an anti-PD-1 based immunotherapy
Cette étude analyse les données de l'essai ayant conduit la "Food and Drug Administration" à autoriser l'utilisation du lifileucel pour traiter les patients atteints d'un mélanome non résécable ou de stade métastatique après l'échec d'une immunothérapie par anti-PD-1
On February 16, 2024, the FDA granted accelerated approval to lifileucel (Amtagvi, Iovance Biotherapeutics, Inc.) indicated for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. Lifileucel is the first tumor-derived T-cell therapy approved by the FDA. In the pivotal cohort of the phase 2 single-arm, trial, Study C-144-01, that served as the basis for approval, the objective response rate (ORR) among patients treated with lifileucel within FDA-approved dose range (7.5 x 109 – 72 x 109 viable cells, n=73) in the primary efficacy cohort was 31.5% (95% CI: 21.1%-43.4%), including 3 (4.1%) complete responses and 20 (27.4%) partial responses. The median duration of response was not reached (95% CI: 4.1 months-not reached). Among the responders (n=23), 56.5%, 47.8%, and 43.5% maintained durable responses at 6, 9, and 12 months, respectively. Among all patients who received lifileucel (N=156), 95.5% of patients experienced at least one Grade 3 treatment-emergent adverse event (TEAE) and 87.8% experienced at least one Grade 4 TEAE. Lifileucel labeling includes a Boxed Warning for treatment-related mortality, prolonged severe cytopenia, severe infection, cardiopulmonary impairment, and renal impairment.
Clinical Cancer Research , résumé, 2025