FDA Approves HEPZATO KIT for Adults Metastatic With Uveal Melanoma

• Approval of Hepzato KIT marks the first approval of a liver-directed therapy for patients with metastatic uveal melanoma.

• The Hepzato KIT is designed to aid doctors in conducting percutaneous hepatic perfusion.

• Hepzato KIT includes an FDA box warning of possible toxicity related to the procedure.

The FDA has granted approval to the HEPZATO KIT (melphalan/Hepatic Delivery System [HDS]). This is a liver-directed treatment for adult patients 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 lung that is amenable to resection or radiation.¹

Results from the phase 3 FOCUS study (NCT02678572) supported the FDA’s decision. In the study, administration of melphalan (Hepzato) was administered using the HDS during a percutaneous hepatic perfusion (PHP) procedure. In 91 patients with hepatic and extrahepatic lesions who were treated every 6 to 8 weeks, for up to 6 treatments, melphalan delivered by HDS led to an objective response rate (ORR) of 36.3% (95% CI, 26.4%-47.0%). Responses included complete response in 7.7% and a 28.6% partial response rate. The median duration of response was 14 months (95% CI, 8.3-17.7), and the disease control rate (DCR) was 73.6% (95% CI, 63.3%-82.3%).

In terms of safety, serious adverse events (AEs) included hemorrhage, hepatocellular injury, and thromboembolic events, occurring in fewer than 5% of patients. Myelosuppressive AEs like thrombocytopenia, anemia, and neutropenia were expected events.

"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," said Gerard Michel, Delcath's chief executive officer, in a 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."

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Focus is a phase 3, single arm, multicenter, open label study. Patients included in the study had either hepatic, or extra-hepatic lesions. Some of the patients were naïve to cancer treatment (56.0%) and others were previously treated (44.0%).^1,2

To be included in the study, patients must have been 18 years of age or older, must weigh 35 kg or greater, have measurable disease in the liver, evidence of limited extrahepatic disease, must not have received chemotherapy, radiotherapy, or chemoembolization within 30 days of treatment, and must have an ECOG performance status of 0 to 1 at screening. Patients with a history of clinically significant pulmonary disease, active bacterial infections, a latex allergy, or prior Whipple’s procedure were not eligible to participate.

"HEPZATO KIT is the only liver-directed therapy that can treat the whole liver," said Vojislav Vukovic, chief medical officer, Delcath, in a press release.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."

_REFERENCES:

_{1. Delcath Systems, Inc. announces FDA Approval of HEPZATO KIT™ for the treatment of adult patients with unresectable hepatic-dominant metastatic uveal melanoma. New release. August 14, 2023. Accessed August 15, 2023.}

_{2. Percutaneous hepatic perfusion in patients with hepatic-dominant ocular melanoma (FOCUS). ClinicalTrials.gov. Updated August 3, 2023. Accessed August 15, 2023. https://bit.ly/3IpfSqo.}