FDA Approves First Tumor-Infiltrating Lymphocyte Therapy to Treat Solid Tumors

The Food and Drug Administration approved the first one-time cell therapy to treat solid tumors and the first tumor-infiltrating lymphocytes (TIL) therapy on Feb. 16. Lifileucel (Amtagvi, Iovance Biotherapeutics, Inc.) is a tumor-derived autologous T-cell immunotherapy approved for adult patients with unresectable or metastatic melanoma. FDA approved the indication through the accelerated approval pathway. The therapy also received Priority Review, Fast Track, Regenerative Medicine Advanced Therapy and Orphan Drug designations.

The agency based its approval on findings from a study of 89 patients with unresectable or metastatic melanoma who had previously been treated with at least one systemic therapy, including a PD-1 blocking antibody, and if BRAF V600 mutation-positive, a BRAF inhibitor with or without a MEK inhibitor.

Among the 73 patients treated with the therapy at the recommended dose, the objective response rate was 31.5%. This included three patients (4.1%) who experienced a complete response and 20 patients (27.4%) with a partial response. Moreover, a significant percentage of patients who responded to the treatment—56.5%, 47.8%, and 43.5%—sustained their responses without tumor progression or mortality at six, nine and 12 months, respectively.

The prescribing information includes a boxed warning for treatment-related mortality, prolonged severe cytopenia, severe infection, as well as cardiopulmonary and renal impairment. Patients receiving lifileucel should be closely monitored before and after infusion for signs and symptoms of adverse reactions.