FDA Approves Hepzato Kit for Metastatic Uveal Melanoma


The Hepzato Kit is a direct-to-liver therapy for patients with uveal melanoma with hepatic metastases.

The Food and Drug Administration (FDA) approved the Hepzato Kit (melphalan/hepatic delivery system) for the treatment of patients with metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases affecting less than 50% of the liver and no extrahepatic (outside of the liver) disease limited to the bone, lymph nodes, subcutaneous tissues or lung that can be surgically removed or treated with radiation.

According to a press release from Delcath Systems, the manufacturer of Hepzato, the company plans to have the kit — which delivers the cancer drug, melphalan, directly to the liver — available toward the end of 2023, with patients continuing to be enrolled in Expanded Access Program sites.

Treatments for liver metastases are needed for this population, as approximately 90% of metastatic uveal melanoma involves the liver, with liver failure often being the cause of death for this patient population, according to the press release. Currently, Hepzato Kit is the only liver-directed therapy for patients with uveal melanoma that has metastasized to the liver.

The National Comprehensive Cancer Network already added percutaneous hepatic perfusion (the procedure used by the Hepzato Kit) for its treatment practice guidelines.

"Hepzato Kit is the only liver-directed therapy that can treat the whole liver," Vojislav Vukovic, chief medical officer at Delcath Systems, said in the release. "Scientific literature supports that Hepzato Kit may have broad applicability in other tumor types, and we intend to expand our development efforts beyond uveal melanoma given the high incidence of unresectable hepatic dominant tumors."

The approval is based on findings from the phase 3 FOCUS study, which included 91 patients with metastatic uveal melanoma who were treated with the Hepzato (melphalan) hepatic delivery system during a percutaneous hepatic perfusion procedure. Fifty-six percent of enrolled patients had no prior treatment, while 44% of patients were previously treated.

Study findings showed that the objective response rate (percentage of patients whose disease shrunk or disappeared after treatment) was 36.3%, with the median duration of response being 14 months. A total of 73.6% of patients experienced disease control, meaning that their cancer shrank or did not grow. There were seven (7.7%) complete responses, where disease is undetectable after treatment, and 26 (28.6%) partial responses, where the disease shrinks but is still visible after therapy.

The Hepzato Kit comes with a boxed warning for patients and clinicians regarding procedure toxicity and myelosuppression (thrombocytopenia, anemia and neutropenia). The warning comes with a Risk Evaluation and Mitigation Strategy program to manage and mitigate those side effects.

Other serious side effects include hemorrhage, liver injury and thromboembolic events, which occurred in 5% or less of treated patients.

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