FDA Approves HEPZATO KIT for Uveal Melanoma with Unresectable Hepatic Metastases

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By Dr. Anish Shah

Bronx-Lebanon Hospital


On August 14, 2023, the U.S. Food and Drug Administration approved HEPZATO KIT – which is melphalan, as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to specific regions.


Study ID number: NCT02678572


Approval was based on the FOCUS study, a single-arm, multicenter, open-label trial that included 91 patients with uveal melanoma with unresectable hepatic metastases restricted to 50% or limited extrahepatic disease – bone, subcutaneous sites, lymph nodes, or lungs – which was amenable to resection or radiation therapy. HEPZATO uses a hepatic delivery system to inject the chemotherapy drug melphalan into the liver, a procedure referred to as percutaneous hepatic perfusion (PHP), administered every 6 to 8 weeks for up to 6 total infusions. The recommended dose was 3 mg/kg based on ideal body weight, with a maximum dose of 220 mg during a single treatment.


Efficacy was established on objective response rate (ORR) and duration of response (DoR). The study demonstrated an ORR of 36.3% (95% CI: 26.4, 47) and a median DoR of 14 months (95% CI: 8.3, 17.7).


Common adverse reactions included thrombocytopenia, fatigue, anemia, nausea, musculoskeletal pain, and several others. There are also severe peri-procedural complications, including hemorrhage, hepatocellular injury, and thromboembolic events.


HEPZATO KIT, designed for liver-directed treatment in uveal melanoma patients with specific conditions, showed an ORR of 36.3% and a median DoR of 14 months. Administered through the Hepatic Delivery System, the recommended dose is 3 mg/kg based on ideal body weight.

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