FDA Approves Hepzato Kit for Metastatic Uveal Melanoma With Liver Metastases


The FDA has approved a liver-directed therapy to treat metastatic uveal melanoma with hepatic metastases.

The FDA has approved a liver-directed melphalan delivery system (Hepzato Kit) to treat patients with unresectable hepatic-dominant metastatic uveal melanoma. Patients must have unresectable hepatic metastases affecting less than 50% of the liver; no extrahepatic disease; or extrahepatic disease that is limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation, to be eligible for treatment.

The approval is supported by data from the phase 3 FOCUS study (NCT02678572). In this single-arm, multicenter, open-label study, patients (n = 91) received melphalan through a hepatic delivery system during a percutaneous hepatic perfusion every 6 to 8 weeks, for up to 6 cycles. The overall response rate was 36.3% (95% CI, 26.4-47.0) and the median duration of response was 14 months (95% CI, 8.3-17.7). There was a 73.6% disease control rate (95% CI; 63.3-82.3). Seven patients (7.7%) achieved a complete response and 26 achieved partial responses (28.6%).

"FDA approval of Hepzato Kit marks the beginning of a new chapter for Delcath and the culmination of the company's commitment to bring this treatment option to patients suffering from metastatic uveal melanoma," Gerard Michel, chief executive officer at Delcath, said in press release. "We look forward to partnering with cancer centers across the country to build a network of treatment sites trained in the use of this novel therapy."

According to the manufacturers, there are about 1,000 cases of metastatic uveal melanoma each year in the United States. Ninety percent of these cases lead to hepatic involvement, and liver failure is often cited as the cause of death. Of note, the National Comprehensive Cancer Network (NCCN) guidelines already support liver-directed therapies for this patient population.

Both patients with hepatic and extra-hepatic lesions were included in the FOCUS study. The trial also included both treatment naïve (56.0%) and pretreated (44.0%) individuals, irrespective of HLA genotype.

The prescribing information for the kit includes a boxed warning with 3 sections. It warns of toxicity related to the procedure, myelosuppression, and a Risk Evaluation and Mitigation Strategy (REMS) program to manage and mitigate these potential toxicities.

Serious adverse events (AEs) were reported in less than 5% of patients, these included hemorrhage, hepatocellular injury, and thromboembolic events.Myelosuppressive AEs that occurred—including thrombocytopenia, anemia, and neutropenia—were reported to be manageable through standard supportive care measures.

"Hepzato Kit is the only liver-directed therapy that can treat the whole liver," Vojislav Vukovic, chief medical officer at Delcath, added. "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."


Delcath systems, inc. announces FDA approval of HEPZATO KIT™ for the treatment of adult patients with unresectable hepatic-dominant metastatic uveal melanoma. Cision newswire. August 14, 2023. Accessed August 15, 2023. https://www.prnewswire.com/news-releases/delcath-systems-inc-announces-fda-approval-of-hepzato-kit-for-the-treatment-of-adult-patients-with-unresectable-hepatic-dominant-metastatic-uveal-melanoma-301900346.html

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