FDA Approves Hepzato Kit for Liver-Directed Treatment of Metastatic Uveal Melanoma


Melphalan/hepatic delivery system (Hepzato Kit; Delcath Systems Inc) is currently the only liver-directed therapy approved by the FDA for the treatment of netastatic uveal melanoma and percutaneous hepatic perfusion.

The FDA has approved melphalan/hepatic delivery system (Hepzato Kit; Delcath Systems Inc) as a liver-directed treatment for adults with metastatic uveal melanoma (mUM) with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lungs that is amenable to resection or radiation, according to a statement from Delcath Systems.1

Ocular Melanoma phrase on the screen. Neurologist use cell technologies at office. | Image Credit: Yurii Kibalnik - stock.adobe.com

Yurii Kibalnik - stock.adobe.com

"[The] 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 [mUM]," Gerard Michel, CEO of Delcath Systems, said in a statement.1 "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."’

In a review published in Current Opinion in Ophthalmology, the authors stated that there have been significant improvements to liver-directed therapy, including systemic chemotherapy and immunotherapy, which has helped life expectancy. The first systemic immunotherapy for mUM, tebentafusp, was approved in 2022.2

National Comprehensive Cancer Network guidelines recommend therapies directed at the liver for those with mUM who have liver metastases. Hepzato Kit is currently the only liver-directed therapy approved by the FDA for the treatment of mUM and percutaneous hepatic perfusion, according to the statement.1

The FDA approval was based on results from the phase 3 single arm, multicenter, open label FOCUS Study (NCT02678572). Hepzato was administered via a hepatic delivery system during a percutaneous hepatic perfusion, according to the statement.1

Investigators included 91 individuals who received treatment every 6 to 8 weeks for up to 6 treatments. Individuals in the study had hepatic and extra-hepatic lesions with a treatment plan. Approximately 56% of individuals were treatment-naïve and 44% were previously treated.1

Objective response rate and duration of response were the main efficacy points at 36.3% and 14 months, respectively.Investigators also determined that the Disease Control Rate observed in patients who were treated was 73.6%, with 7.7% being complete responses and 28.6% being partial responses.1

"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,” Vojislav Vukovic, MD, PhD, MSc, chief medical officer at Delcath Systems, said in a statement.1

The boxed warnings include 3 sections, including toxicities related to the procedure, myelosuppression, and a Risk Evaluation Mitigation Strategy program. Serious adverse events (AEs) associated with the kit included hemorrhage, hepatocellular injury, and thromboembolic events, which occurred in less than 5% of individuals in the study, according to the statement.1

Furthermore, myelosuppressive AEs included thrombocytopenia, anemia, and neutropenia, which investigators said are known and predictable AEs associated with melphalan and are managed with standard supportive care measures.1

Delcath Systems plans to make the product available in the fourth quarter of 2023 with patients continuing to be enrolled and treated at Expanded Access Program sites.1


  1. Delcath Systems Inc announces FDA approval of Hepzato Kit for the treatment of adult patients with unresectable hepatic-dominant metastatic uveal melanoma. News release. PR 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
  2. Reichstein D, Brock A, Lietman C, McKean M. Treatment of metastatic uveal melanoma in 2022: improved treatment regimens and improved prognosis. Curr Opin Ophthalmol. 2022;33(6):585-590. doi:10.1097/ICU.0000000000000905
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