FDA Completes Review of Hepzato Combo in HER2– Metastatic Breast Cancer

The FDA finished its 30-day review of Hepzato with standard care in liver-dominant metastatic breast cancer.

Among patients with liver-dominant metastatic breast cancer, the United States Food and Drug Administration (FDA) has completed its 30-day review of an investigational new drug application for Hepzato in combination with standard of care, according to a news release from Delcath Systems, Inc.

The Hepzato combination is currently being evaluated in a phase 2 clinical trial, according to the news release, which went on to note that the Company is now cleared to initiate patient enrollment in the United States with the FDA's review complete.

“This randomized phase 2 trial marks an important milestone as we expand the clinical investigation of Hepzato into patients with liver-dominant metastatic breast cancer,” said Gerard Michel, chief executive officer of Delcath Systems, Inc. “We are excited to bring new hope to patient populations in indications beyond metastatic uveal melanoma and to further demonstrate the potential of Hepzato to address unmet needs in oncology. This study underscores our commitment to broadening the applications of Hepzato and the underlying hepatic delivery system, positioning us as a platform technology that can offer directed treatment options for a variety of liver-dominant cancers.”

The phase 2 trial will evaluate the safety and efficacy of Hepzato plus standard of care, as well as compare the investigative combination with standard of care alone in patients with liver-dominant HER2-negative metastatic breast cancer following disease progression with other therapeutic options. Standard of care options include physician’s choice of eribulin, vinorelbine or capecitabine, notably.

Additionally, the news release says that approximately 90 patients are expected to be enrolled in the randomized, controlled trial. Patient enrollment for the trial is expected to begin in the fourth quarter of 2025, with more than 20 sites across the United States and Europe participating.

Data on hepatic progression-free survival, which serves as the trial's primary end point, is expected to be announced by the end of 2028, while results for the secondary endpoint of overall survival are expected in 2029.

More Information on Metastatic Breast Cancer and Hepzato

HER2-negative metastatic breast cancer with liver metastases is estimated to affect approximately 7,000 patients each year in the United States, according to the news release, which notes that these patients are candidates for third line treatment. Patients with a significant burden of liver metastases comprise a portion of this patient population which is important to note because a significant burden of liver metastases is likely to be the primary cause of mortality for these individuals. In the news release, the Company states that by focusing on this demographic of patients with unmet needs and limited treatment alternatives, they intend to offer a novel therapeutic option.

Hepzato is an alkylating agent and is currently only approved as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases involving less than 50% of the liver. According to the Hepzato KIT website, the Company states that, to be eligible for treatment, patients must have no extrahepatic disease, or disease outside the liver limited to the bone, lymph nodes, subcutaneous tissue, or lung that is amenable to resection or radiation.

Hepzato is a component of the Hepzato KIT and is delivered via intra-arterial infusion into the hepatic artery, the website continues. The currently recommended dosage for patients who are eligible for treatment is 3 milligrams per kilogram (based on body weight), with a maximum dose of 220 milligrams during a single Hepzato treatment. The drug is given every six to eight weeks and is infused over 30 minutes followed by a 30-minute washout period, but can be postponed based on toxicities, as per clinical judgement.

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