FDA Approval of Percutaneous Hepatic Perfusion Offers Patients New Hope

August 23, 2024

In a significant advancement for the treatment of metastatic uveal melanoma, the U.S. Food and Drug Administration (FDA) recently approved Percutaneous Hepatic Perfusion (PHP) with the HEPZATO KIT. This new therapy represents a breakthrough in the management of this rare but aggressive cancer, offering renewed hope for patients who have historically faced limited options. The recent findings from the Phase 3 FOCUS trial, presented at the American Society of Clinical Oncology (ASCO) annual meeting, underscore PHP's efficacy and potential as a first-line treatment.

A Paradigm Shift in Treatment

Uveal melanoma originates in the eye and affects roughly 4,500 individuals annually. Approximately 50% of these patients will develop metastatic disease, with up to 90% of those metastases predominantly or only affecting the liver. Until recently, treatment options for metastatic uveal melanoma were sparse and often ineffective, making the FDA’s approval of PHP a pivotal moment in oncology.

Phase 3 FOCUS Trial: Key Findings

The Phase 3 FOCUS trial, was a multinational, multi-site trial led by Dr. Jonathan Zager of the Cutaneous Oncology Program at Moffitt in collaboration with Delcath Systems, Inc., has demonstrated that PHP with the HEPZATO KIT is a highly effective treatment for metastatic uveal melanoma to the liver. According to Dr. Jonathan Zager, a surgical oncologist, the data from this trial showed that PHP was more effective than any best alternative care (BAC) and should be considered a first-line treatment for patients with metastatic uveal melanoma.

Dr. Zager highlighted that PHP performed with the HEPZATO KIT has shown superior efficacy compared to the BAC available at the time of the trial. “We should convince the community to use this treatment in the first line, as there is data to support treating patients with a low burden of disease earlier, which will give them the best chances at a response,” Zager stated.

We should convince the community to use this treatment in the first line, as there is data to support treating patients with a low burden of disease earlier will give them the best chances at a response.

Dr. Jonathan Zager

How Percutaneous Hepatic Perfusion Works

PHP involves a sophisticated process where catheters and balloons are used to isolate the liver from the body’s circulatory system. This isolation allows for high-dose chemotherapy to be administered directly to the liver, targeting the tumor more effectively while minimizing systemic exposure to the chemo. The blood exiting the liver is then collected and filtered outside the body to remove most of the residual chemotherapy, further reducing systemic toxicity. PHP can be administered up to six times, typically every six to eight weeks.

Clinical Implications and Future Directions

Since the FDA's approval of the HEPZATO KIT in August 2023, PHP has been incorporated into the standard of care for metastatic uveal melanoma. Moffitt Cancer Center, along with a growing number of institutions, has performed nearly 300 PHP procedures, with 30 cases performed since commercial availability began in early January 2024.

Dr. Zager emphasizes the importance of early administration of PHP. While the treatment has shown good response rates in patients who have failed prior therapies, it is most effective when used as a first-line treatment.

“On the flip side, if we don’t use PHP early in the treatment, there is a chance the patient may become ineligible for PHP due to progression in the liver or metastasis to other sites,” he cautioned.

A recent publication in Annals of Surgical Oncology in August 2024 showed that PHP is more effective as first-line therapy than immunotherapy or other liver-directed therapies. Additionally, according to the data in the manuscript, PHP is also more effective than immunotherapy or other liver-directed therapies as a second line, showing it can be used to salvage first-line therapy failures.

Expanding Access and Enhancing Care

Despite the promising results, there remains a critical need for wider adoption and integration of PHP into clinical practice. Dr. Zager stresses the goal of increasing the number of institutions offering this treatment and ensuring more patients have access to this potentially life-saving therapy. “We have patients coming to us from across the country on a daily basis,” he says.

Conclusion

The approval of Percutaneous Hepatic Perfusion with the HEPZATO KIT marks a transformative development in the treatment of metastatic uveal melanoma. By providing a more effective and targeted treatment option, PHP offers new hope to patients who have long faced limited and often ineffective choices. As healthcare providers, embracing this innovative therapy and staying informed about its application will be crucial in advancing patient care and improving outcomes for those battling this challenging disease.

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