FDA Grants Accelerated Approval to Lifileucel For Treatment of Advanced Melanoma


This marks the first and only time that an individualized T cell therapy has been approved by the FDA for a solid tumor cancer.

The FDA has approved lifileucel (Amtagvi; Iovance Biotherapeutics Inc) as a suspension for intravenous infusion indicated for 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 mitogen-activated protein kinase inhibitor, according to a press release from the company. This marks the first and only time that an individualized T cell therapy has been approved by the FDA for a solid tumor cancer.1

Dermatologist examining moles of patient in clinic | Image Credit: Pixel-Shot - stock.adobe.com

Image Credit: Pixel-Shot - stock.adobe.com

“One-time treatment with [lifileucel] offered clinically meaningful and deep, durable responses in the Phase 2 clinical trial, and I am excited by its potential as a much-needed new treatment option for the many advanced melanoma patients who progress on the current standard of care,” Alexander N. Shoushtari, MD, melanoma oncologist and cellular therapist at Memorial Sloan Kettering Cancer Center, said in the press release.1

Lifileucel is a tumor-derived autologous T cell immunotherapy and has been approved under accelerated approval. The approval was based on the overall response rate (ORR) and duration of response (DOR) from the C-144-01 (NCT02360579) clinical trial, according to the press release. The trial is a global, multicenter trial that investigated the drug in the same patient population, demonstrating deep and durable responses.1

About The Trial

Trial Name: Study of Lifileucel (LN-144), Autologous Tumor Infiltrating Lymphocytes, in the Treatment of Patients With Metastatic Melanoma (LN-144)

ClinicalTrials.gov ID: NCT02360579

Sponsor: Iovance Biotherapeutics Inc

Estimated Completion Date: January 2025

The primary efficacy analysis included 74 individuals from cohort 4 who received the drug, 31.5% of whom achieved an ORR, evaluated by the Response Evaluation Criteria in Solid Tumors (RECIST 1.1), according to the press release. Furthermore, the median duration of response has not be reached at approximately 18.6 months of follow-up. Additionally, approximately 43.5% of responses had a duration of response longer than 12 months, according to the results.1

In a pooled efficacy set including 153 patients in total from cohort 4 and cohort 2, 31.4% achieved an objective response by RECIST 1.1 and 54.2% of responses had a duration greater than 12 months, according to the press release. Further, the median DOR has not yet been reached at 21.5 months of follow-up.1

“The approval of [lifileucel] offers hope to those with advanced melanoma who have progressed following initial standard of care therapies, as the current treatment options are not effective for many patients,” Samantha R Guild, JD, president of AIM at Melanoma Foundation, said in the press release. “This one-time cell therapy represents a promising innovation for the melanoma community, and we are excited by its potential to transform care for patients who are in dire need of additional therapeutic options.”

Lifileucel is for autologous use only, with boxed warning including treatment-related mortality, prolonged server cytopenia, severe infection, and cardiopulmonary and renal impairment, according to the press release. The full study results of the C-144-01 trial were published in The Journal for ImmunoTherapy of Cancer.1,2

  1. Iovance’s Amtagvi (lifileucel) receives US FDA accelerated approval for advanced melanoma. News release. Iovance. February 16, 2024. Accessed February 16, 2024. https://ir.iovance.com/news-releases/news-release-details/iovances-amtagvitm-lifileucel-receives-us-fda-accelerated
  2. Chesney J, Lewis KD, Kluger H, Hamid O, et al. Efficacy and safety of lifileucel, a one-time autologous tumor-infiltrating lymphocyte (TIL) cell therapy, in patients with advanced melanoma after progression on immune checkpoint inhibitors and targeted therapies: pooled analysis of consecutive cohorts of the C-144-01 study. J Immunother Cancer. 2022;10(12):e005755. doi:10.1136/jitc-2022-005755
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