One-Time Cell Therapy Amtagvi Gets Accelerated Approval for Advanced Melanoma

The Food and Drug Administration (FDA) has granted accelerated approval to Amtagvi™ (lifileucel) for the treatment of adults 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.

Amtagvi is a tumor-derived autologous T-cell immunotherapy. While the exact mechanism of action is unknown, Amtagvi is designed to deploy patient-specific T cells called tumor infiltrating lymphocyte (TIL) cells to locate and attack cancer cells.

The approval was based on data from the phase 2 C-144-01 study (ClinicalTrials.gov Identifier: NCT02360579), which evaluated lifileucel in adults with unresectable or metastatic melanoma who were previously treated with at least 1 systemic therapy, including a PD-1 blocking antibody, and if BRAF V600 mutation-positive, a BRAF inhibitor or BRAF inhibitor with or without MEK inhibitor. 

The primary efficacy analysis included 73 patients who received lifileucel within the recommended dosing range of 7.5 x 10⁹ to 72 x 10⁹ viable cells; all patients had received prior anti-PD-(L)1 therapy, 63 received prior anti-CTLA-4 therapy, 42 received anti-PD1/anti-CTLA-4 combination therapy and 20 received a BRAF inhibitor or combination therapy with BRAF and MEK inhibitors. 

Among the 73 patients, the objective response rate was 31.5% (95% CI, 21.1-43.4), with 3 complete responses and 20 partial responses. Median time to initial response was 1.5 months. Median duration of response was not reached, with 56.5%, 47.8%, and 43.5% of responders having responses lasting for at least 6, 9, and 12 months, respectively.

The prescribing information for Amtagvi includes a Boxed Warning associated with treatment-related mortality, prolonged severe cytopenia, severe infection, cardiopulmonary, and renal impairment. The most common non-laboratory adverse reactions (incidence at least 20%) were chills, pyrexia, fatigue, tachycardia, diarrhea, febrile neutropenia, edema, rash, hypotension, alopecia, infection, hypoxia, and dyspnea.

Amtagvi is supplied in 1 to 4 infusion bag(s), with each bag containing approximately 100mL to 125mL of frozen suspension of tumor-derived T cells. Amtagvi should be administered in an inpatient hospital setting with an intensive care facility. Treatment is administered as a single dose for infusion, with each dose containing 7.5 x 10⁹ to 72 x 10⁹ viable cells. 

“One-time treatment with Amtagvi 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,” said Dr Alexander N. Shoushtari, Melanoma Oncologist & Cellular Therapist at Memorial Sloan Kettering Cancer Center. 

The Company is offering a comprehensive support program called IovanceCares for eligible patients to receive Amtagvi therapy.

This article originally appeared on MPR