Delcath’s Hepzato Kit Receives FDA Approval for the Treatment of Metastatic Uveal Melanoma

August 14, 2023—Delcath Systems, Inc., an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, announced that the FDA approved the company’s Hepzato Kit (melphalan/hepatic delivery system).

Hepzato Kit is indicated for liver-directed treatment for adult patients with metastatic uveal melanoma (mUM) with unresectable hepatic metastases affecting < 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.

The company noted that National Comprehensive Cancer Network (NCCN) guidelines recommend liver-directed therapies for mUM patients with liver metastases. Delcath’s FDA-approved Hepzato Kit liver-directed therapy enables percutaneous hepatic perfusion (PHP), which is included in the NCCN guidelines.

The company plans to have commercial product available in the fourth quarter of 2023, and patients will continue to be enrolled and treated at Expanded Access Program sites.

According to Delcath, the FDA approval of Hepzato Kit was based primarily on the results of the FOCUS study, which is a phase 3, single-arm, multicenter, open-label study that administered Hepzat (melphalan) via the hepatic delivery system during a PHP procedure.

The patient population enrolled in the FOCUS study included patients with hepatic and extra-hepatic lesions subject to a treatment plan, as well as both treatment-naïve (56%) and previously treated (44%) patients, irrespective of HLA genotype.

In the FOCUS study, 91 patients received treatment every 6 to 8 weeks, for up to 6 treatments. The main efficacy endpoints were objective response rate (ORR) and duration of response (DoR), as assessed by an independent review committee using RECIST v1.1.

As reported in the company’s press release, ORR was 36.3% (95% CI, 26.4-47.0) and median DoR was 14 months (95% CI, 8.3-17.7). The disease control rate observed in treated patients was 73.6% (95% CI, 63.3-82.3), with seven complete responses (7.7%) and 26 (28.6%) partial responses.

The company advised that the Hepzato Kit prescribing information has a boxed warning, which includes three sections: toxicity related to the procedure, myelosuppression, and a Risk Evaluation and Mitigation Strategy (REMS) program to manage and mitigate these risks. The Hepzato Kit REMS is designed to ensure consistent conduct of the PHP procedure and that only treatment teams who have received appropriate training perform the PHP procedure.

“Hepzato Kit is the only liver-directed therapy that can treat the whole liver,” stated Delcath’s Chief Medical Officer Vojislav Vukovic, MD, in the press release. “Scientific literature supports that Hepzato Kit may have broad applicability in other tumor types, and we intend to expand our development efforts beyond uveal melanoma given the high incidence of unresectable hepatic dominant tumors.”

Delcath also announced that the FDA approval effectively triggers the second tranche of financing tied to the Private Investment in Public Equity (PIPE) financing, which was announced on March 29, 2023. Participants in the PIPE have 21 days to exercise their Tranche A warrants, translating to up to approximately $34.9 million of additional funding to Delcath. In addition, upon the company’s announcement of recording at least $10.0 million in quarterly United States revenue from the commercialization of Hepzato Kit, participants in the PIPE will have 21 days to exercise their Tranche B warrants, resulting in up to an additional $24.9 million in funding to Delcath.