The US Food and Drug Administration (FDA) has approved the Hepzato Kit (melphalan/hepatic delivery system) as a liver-directed treatment for adults with metastatic uveal melanoma and unresectable hepatic metastases affecting less than 50% of the liver. Patients must have no extrahepatic disease or have extrahepatic disease that is limited to the bone, lymph nodes, subcutaneous tissues, or lung and is amenable to resection or radiation.
The approval was based on data from the phase 3 FOCUS study (ClinicalTrials.gov Identifier: NCT02678572), which was designed to evaluate the Hepzato Kit during a percutaneous hepatic perfusion procedure in 91 patients with hepatic-dominant uveal melanoma.
Patients received 3 mg/kg of melphalan administered intraarterially using the hepatic delivery system every 6 to 8 weeks for up to 6 infusions.
The objective response rate was 36.3%, the complete response rate was 7.7%, and the partial response rate was 28.6%. The median duration of response was 14 months. The disease control rate was 73.6%.
The most common adverse reactions (≥20%) were 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.
The prescribing information for the Hepzato Kit includes a boxed warning regarding the risks of severe peri-procedural complications and myelosuppression. Due to these risks, the Hepzato Kit is only available through a risk evaluation and mitigation strategy program.
The Hepzato Kit is supplied with the Hepzato 5×5 Drug Pack and the hepatic delivery system. Each 5×5 Hepzato Kit Drug Pack includes Hepzato (mephalan) and diluents for reconstitution and dilution. The product is expected to be available in the fourth quarter of 2023.
Delcath Systems, Inc. announces FDA approval of Hepzato Kit™ for the treatment of adult patients with unresectable hepatic-dominant metastatic uveal melanoma. News release. Delcath Systems, Inc. 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.
Hepzato Kit. Package Insert. Delcath Systems, Inc; 2023. Accessed August 15, 2023. https://hepzatokit.com/wp-content/uploads/2023/08/Hepzato-Kit-Prescribing-Information.pdf.
This article originally appeared on MPR