The Food and Drug Administration announced yesterday the approval of HEPZATO Kit to treat ocular melanoma that has spread to the liver. HEPZATO uses a hepatic delivery system to inject the chemotherapy drug melphalan into the liver, a procedure referred to as percutaneous hepatic perfusion (PHP).
Moffitt Cancer Center’s Jonathan S. Zager, M.D., was the lead international principal investigator on the multinational FOCUS phase 3 clinical trial to test the procedure, which is manufactured by Delcath Systems, Inc.
This is a breakthrough therapy that will help a patient population that has very few treatment options.The phase 3 trial was very positive in terms of overall response rate and durability of the responses, with very manageable toxicity.Jonathan S. Zager, M.D. Chief academic officer and surgical oncologist in the Department of Cutaneous Oncology at Moffitt.
Unlike traditional chemotherapy that circulates throughout the entire body, percutaneous hepatic perfusion uses a series of catheters and balloons to isolate the liver from the body’s circulatory system, allowing for high dose chemotherapy to be delivered directly to the liver only.
The blood is collected as it exits the liver and filtered outside the body via a bypass circuit to remove any remaining chemo. Patients can receive percutaneous hepatic perfusion up to six times, usually performed six to eight weeks apart.
Uveal melanoma is a cancer that originates in the eye. Roughly 4,500 people receive the diagnosis each year. About half of those patients will develop metastatic disease, with 90% of those metastases being solely or predominately in the liver.
Moffitt led the pivotal international trial, which began in 2016 and included 40 institutions in the U.S. and Europe. The trial accrued 102 patients to the percutaneous hepatic perfusion arm. In June 2022, Zager shared final phase 3 results at the American Society of Clinical Oncology Annual Meeting.
The objective response rate, meaning evidence tumors are responding to therapy, for PHP patients was almost triple compared to the best alternative care options (35.2% vs. 12.5%). And 8% of the patients in the PHP arm of the trial had a complete response to therapy versus none in the best alternative care group. Progression free survival was 9.03 months in PHP vs. 3.12 months in the best alternative care group.Jonathan S. Zager, M.D. Chief academic officer and surgical oncologist in the Department of Cutaneous Oncology at Moffitt.
Moffitt enrolled and treated the first and last patients on the FOCUS phase 3 trial and has performed the procedure nearly 200 times to date.
We are very grateful to the patients who participated in the clinical trials. This approval has been years in the making. I am grateful to have had the opportunity to be the lead principal investigator on the trial and super excited to offer this therapy to more patients with metastatic uveal melanoma.Jonathan S. Zager, M.D. Chief academic officer and surgical oncologist in the Department of Cutaneous Oncology at Moffitt