News|Articles|January 2, 2026

Hepzato Kit Safe, Effective in Metastatic, Unresectable Uveal Melanoma

Author(s)Alex Biese
Fact checked by: Spencer Feldman
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Key Takeaways

  • The Hepzato Kit is the first FDA-approved liver-directed therapy for unresectable metastatic uveal melanoma, showing consistent efficacy across diverse patient subgroups.
  • Patients with lower tumor burden experienced significantly higher objective response rates and longer progression-free survival and overall survival.
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Subgroup analysis of the phase 3 FOCUS study of patients with uveal melanoma has been announced.

Among patients with unresectable metastatic uveal melanoma, the drug/device combination of Hepzato Kit was found to be safe and effective, according to subgroup analysis of the phase 3 FOCUS study announced in a news release issued by Delcath Systems, Inc., the interventional oncology company behind the Hepzato Kit, and detailed in an article published in the Journal of Cancer Research and Clinical Oncology.

The Hepzato Kit, as noted in the news release, is currently the only liver-directed treatment that has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with unresectable metastatic uveal melanoma.

The prespecified analysis of clinically relevant subgroups included 91 patients who were treated as part of the FOCUS study, with findings including that consistent tumor response was observed regardless of patients’ age, sex, geographic region, the presence or absence of extrahepatic lesions, and prior therapy status.

Researchers found there to be a significantly higher objective response rate for patients with tumor burden below the median (51.1% versus 22.2%), with no other significant differences between subgroups, while there was also a significantly longer median progression-free survival for patients with tumor burden below the median (11.3 months versus 5.8 months), with no other significant differences between subgroups.

Glossary

Objective response rate (ORR): how many patients’ tumors shrink or disappear after treatment.

Progression-free survival (PFS): the length of time during and after treatment that a patient lives without the cancer growing or spreading.

Overall survival (OS): the length of time patients are alive after starting a treatment.

Regarding overall survival, there was a significantly longer median overall survival for patients with a smaller extent of tumor liver involvement (22.4 months versus 16.8 moths), tumor burden below the median (26.7 months versus 15.4 months) and low/normal LDH (23.4 months versus 15.3 months). And, of the 33 patients who achieved a complete response or partial response, 57.6%, or 19 patients, responded within the first or second treatment cycle, and 33.3%, or 11 patients, responded in the fourth through sixth cycles.

The news release further reported that the overall safety profile of the Hepzato Kit was found to be similar across subgroups, with no evidence of cumulative toxicity with successive treatment cycles. The incidence of serious side effects and grade 3 (severe) or 4 (life-threatening) side effects was consistent with the overall study population, and the news release noted that no treatment-related deaths happened.

“These subgroup analyses provide valuable insights into optimizing treatment with Melphalan/HDS for patients with unresectable metastatic uveal melanoma, underscoring the importance of early intervention in patients with lower tumor burden to maximize clinical benefits,” said Dr. Vojislav Vukovic, Chief Medical Officer of Delcath, in a statement included in the news release. “The consistent efficacy and manageable safety profile across diverse patient groups further validate this liver-directed therapy as a key option in managing this challenging disease.”

The Hepzato Kit (Hepzato (melphalan) for injection/hepatic delivery system) is designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects during a PHP procedure, as explained in the news release.

The FDA approved the Hepzato Kit in August 2023, marking the first direct-to-liver therapy available for patients with metastatic uveal melanoma (also referred to as ocular melanoma) with unresectable liver metastases.

“This new therapy is really a big deal,” Dr. Richard Carvajal, deputy physician and chief overseeing medical oncology and hematology at the Northwell Health Cancer Institute, previously said in an interview with CURE. “The disease we’re dealing with is a rare type of melanoma — there are only a few thousand cases a year diagnosed in the United States, and before this approval, there was only one other treatment that was approved.”

Uveal melanoma, as explained by the National Cancer Institute on its website, is a rare cancer that begins in the cells that make the dark-colored pigment, called melanin, in the uvea or uveal tract of the eye. Uveal melanoma is the most common type of eye cancer in adults, and it is also called intraocular melanoma. Metastatic cancer is disease that has spread from its original site, and unresectable disease is disease that cannot be removed by surgery.

References

  1. “Delcath Systems Announces Publication of Subgroup Analyses of the Phase 3 FOCUS Study of Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma,” news release; https://www.businesswire.com/news/home/20251231288521/en/Delcath-Systems-Announces-Publication-of-Subgroup-Analyses-of-the-Phase-3-FOCUS-Study-of-MelphalanHepatic-Delivery-System-in-Patients-with-Unresectable-Metastatic-Uveal-Melanoma
  2. “Uveal melanoma,” National Cancer Institute; https://www.cancer.gov/publications/dictionaries/cancer-terms/def/uveal-melanoma
  3. “Hepzato Kit’s Approval Is a ‘Big Deal’ for Metastatic Uveal Melanoma,” CURE; https://www.curetoday.com/view/hepzato-kit-s-approval-is-a-big-deal-for-metastatic-uveal-melanoma

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