Hepzato FDA Approval History

Last updated by Judith Stewart, BPharm on Aug 31, 2023.

FDA Approved: Yes (First approved August 14, 2023)
Brand name: Hepzato
Generic name: melphalan
Dosage form: Lyophilized Powder for Injection
Company: Delcath Systems, Inc.
Treatment for: Uveal Melanoma

Hepzato (melphalan) is an alkylating drug used as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases.

• Uveal melanoma is a very rare form of cancer that affects melanocytes in the eye. About half of all patients with uveal melanoma will develop metastatic disease, with the metastases occurring predominantly in the liver (~90% of patients), and less commonly in the lungs and bones.
• 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.
• Hepzato contains melphalan, a well-known chemotherapeutic agent that has been used for decades in the treatment of multiple myeloma and ovarian cancer. Melphalan works by alkylating guanine to inhibit DNA and RNA synthesis, causing cytotoxicity in the tumor cells.
• The Hepzato Kit is a combination product that administers Hepzato directly to the liver through Delcath's novel device delivery system, the Hepatic Delivery System (HDS), which permits higher drug exposure in target tissues while limiting systemic toxicity via a procedure called percutaneous hepatic perfusion (PHP). During PHP, catheters are placed percutaneously and the liver is temporarily isolated from the body’s circulatory system, during which time a 30 minute infusion of Hepzato directly to the liver occurs. The blood is filtered as it leaves the liver, and is then returned to systemic circulation.
• Hepzato is administered by intraarterial infusion into the hepatic artery over 30 minutes followed by a 30- minute washout period. Treatments should be administered every six to eight weeks but can be delayed until recovery from toxicities and as per clinical judgement.
• The Hepzato product label carries a Boxed Warning for severe peri-procedural complications (including hemorrhage, hepatocellular injury, and thromboembolic events) and myelosuppression. Hepzato is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the Hepzato Kit REMS.
• Warnings and precautions associated with Hepzato include hypersensitivity reactions, gastrointestinal disturbances, carcinogenic/mutagenic effects, embryo-fetal toxicity, and infertility.
• Common adverse reactions or laboratory abnormalities include thrombocytopenia, fatigue, anemia, nausea, musculoskeletal pain, leukopenia, abdominal pain, neutropenia, vomiting, increased alanine aminotransferase, prolonged activated partial thromboplastin time, increased aspartate aminotransferase, increased alkaline phosphatase, and dyspnea.

Development timeline for Hepzato

Further information

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