What Melanoma Patients Need to Know about Hepzato Kit

You may have heard of Hepzato Kit, also known as Liver-Directed Melphalan, the first liver-directed therapy available for patients with metastatic uveal melanoma who have liver metastases that cannot be surgically removed.

While uveal melanoma originates in the eye, new therapies like Hepzato Kit are important because the liver is the most common site of metastasis.

If you have questions about Hepzato Kit, keep reading: 

What is Hepzato Kit?

Hepzato Kit is a novel therapy that delivers melphalan, a well-studied chemotherapy drug first approved in 1964, directly to the liver through a novel delivery system called the Hepatic Delivery System (HDS). This allows oncologists to treat metastatic uveal melanoma liver tumors with much higher doses of melphalan than would otherwise be possible. By administering the therapy only to the liver, systemic side effects are significantly reduced. 

What does the research say about Hepzato Kit?

Hepzato Kit was approved based on results from Delcath's Phase 3 FOCUS clinical trial. In this single arm, multicenter study, 91 patients with metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases received Hepzato Kit every 6 to 8 weeks, for up to 6 treatments. Together, these patients had an objective response rate (ORR) – meaning the percentage of patients that had a significant reduction in size or disappearance of their tumor – of 36.3% and a median duration of response (DoR) of 14 months. The Disease Control Rate (DCR) – meaning the percentage of patients whose tumors either shrink or remain stable – among the patient cohort was 73.6% and included 7 complete responses (7.7%) and 26 partial response (28.6%).¹

The study enrolled patients with metastatic uveal melanoma with metastases predominately involving the liver (liver dominant). Patients with limited metastases beyond the liver, such as in the bone, subcutaneous sites, lymph nodes, or lung were permitted if the life-threatening component of the uveal melanoma was in the liver and the tumors outside the liver had a defined treatment plan. Fifty six percent of patients in the cohort had not received any previous systemic treatment for their melanoma.  

How does Hepzato Kit treat advanced uveal melanoma?

Hepzato Kit helps patients with uveal melanoma live longer by shrinking  melanoma tumors that have spread to the liver and cannot be surgically removed, thereby preserving organ function. Melphalan, the medicine used in Hepzato Kit, inhibits the proliferation of cancer cells by damaging DNA and interfering with its replication.

All melanoma treatments, including Hepzato Kit, have side effects, which can sometimes be serious. Patients should talk with their physician to learn more about the side effects of Hepzato Kit and other melanoma treatment options.

How does Hepzato Kit compare against existing melanoma therapies?

Cancer specialists can treat many types of cancer, including metastatic uveal melanoma, with medications that kill fast-growing cells in your body called chemotherapies. Chemotherapies are not often used in the treatment of cutaneous melanoma, they are re-emerging as a potential tool in the treatment of uveal melanoma. 

Who is eligible to receive Hepzato Kit?

The U.S. Food and Drug Administration (FDA) approved Hepzato Kit for the treatment of patients with:

• Metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases affecting less than 50% of the liver with no extrahepatic (outside of the liver) metastases, and
• Metastatic uveal melanoma with unresectable (ineligible for surgery) liver metastases affecting less than 50% of the liver with limited metastases beyond the liver, such as in the bone, subcutaneous sites, lymph nodes, or lung that is amenable to treatment with surgery or radiation.

Speak to your doctor to see if you are eligible to be treated with Hepzato Kit.

How is Hepzato Kit administered? 

Patients receive liver-directed melphalan (Hepzato Kit) directly into the hepatic artery (the main source of blood to the liver) through a minimally invasive procedure known as percutaneous hepatic perfusion (PHP). During this procedure, the liver is temporarily isolated from the body’s circulatory system during which melphalan is infused directly to the liver over a 30-minute period. Upon exiting the liver, the patient’s blood is filtered before returning it to systemic circulation.

Treatments are administered in a hospital setting at select sites every six to eight weeks. 

What do the experts say about Hepzato Kit?

“This new therapy is really a big deal,” said Dr. Richard Carvajal, Deputy Physician-In-Chief and Director of Medical Oncology at the Northwell Health Cancer Institute. “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.”

“This novel therapy delivers a chemotherapy drug directly to the liver and addresses an important unmet need for patients with uveal melanoma that has spread to the liver, the predominant organ of metastasis,” said MRA Chief Science Officer Joan Levy, PhD. “Further, it showcases the ingenuity and dedication of melanoma researchers who contributed towards its development and patients who participated in clinical trials testing this unique treatment approach. It’s proof that we can develop meaningful therapies for patients with rare diseases that can improve patient survival and quality of life.”