FDA Approves Hepzato (melphalan) for Uveal Melanoma with Hepatic Metastases

Photo by National Cancer Institute / Unsplash

FDA Published June 2, 2026 Medically reviewed June 2, 2026 DOI ↗ By Dr. Julia Lee, PhD · Oncology, Genomics & Drug Development

Key Takeaway

Consider Hepzato for liver-directed treatment in uveal melanoma patients with limited hepatic metastases and no or resectable extrahepatic disease.

The FDA has approved Hepzato (melphalan) for injection as a liver-directed treatment for adult patients with uveal melanoma who have 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. Hepzato is administered via intra-arterial infusion into the hepatic artery using the Hepzato Kit Hepatic Delivery System (HDS). This approval provides a targeted treatment option for patients with liver metastases from uveal melanoma, a rare and aggressive cancer with limited therapeutic options. The recommended dose is 3 mg/kg based on ideal body weight, with a maximum of 220 mg per treatment, given every 6 to 8 weeks for up to 6 infusions. Clinicians must complete Hepzato Kit REMS training before administration.

＋ Clinical Details (Mechanism · Dosing · Trial Data · Warnings)

Mechanism of Action

Melphalan is an alkylating drug that cross-links DNA, inhibiting DNA replication and transcription, leading to cell death.

Indication & Patient Population

Hepzato is indicated 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 the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.

Dosing & Administration

The recommended dose is 3 mg/kg based on ideal body weight (IBW), with a maximum absolute dose of 220 mg per treatment. IBW is calculated per Table 1 in the label. Hepzato is administered by intra-arterial infusion into the hepatic artery over 30 minutes followed by a 30-minute washout period. Treatments are given every 6 to 8 weeks for up to 6 total infusions. Dosage reduction to 2 mg/kg is recommended for Grade 4 neutropenia >5 days despite growth factor support or with neutropenic fever, or Grade 4 thrombocytopenia >5 days or with hemorrhage requiring transfusion. Discontinue if life-threatening or persistent toxicity not resolved to Grade 2 or less by 8 weeks.

Key Clinical Trial Data

Trial data not available in label.

Warnings & Contraindications

Contraindicated in patients with hypersensitivity to melphalan or natural rubber latex (the double balloon catheter contains latex). Discontinue oral anticoagulation and drugs affecting platelet function prior to procedure. Discontinue ACE-inhibitors, calcium channel blockers, or alpha-1-adrenergic blockers prior to procedure. Conduct baseline hematologic testing: hemoglobin ≥10 g/dL, platelets ≥100,000/microliter, neutrophils >2000/microliter. Hepzato is a hazardous drug; follow special handling and disposal procedures.

Place in Therapy

Hepzato provides a liver-directed treatment option for patients with uveal melanoma and hepatic metastases meeting specific criteria. It is administered via the Hepzato Kit Hepatic Delivery System, requiring specialized training and REMS certification.

Study Details

Study typeFda approval

PublishedAug 2023

View Original Abstract ↓

1 INDICATIONS AND USAGE HEPZATO for injection, as a component of the HEPZATO KIT, is indicated 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 the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation. HEPZATO is an alkylating drug indicated 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 the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.( 1 )