Delcath Systems’ Hepzato Kit (melphalan/hepatic delivery system) has been approved by the US Food and Drug Administration (FDA) for treating metastatic uveal melanoma.

Uveal melanoma is an ophthalmic cancer with a high chance of metastasis. The Hepzato Kit specifically focuses on treating the extra-ocular metastases of uveal melanoma, which affect either up to 50% of the liver tissue without extrahepatic disease, or metastases limited to extrahepatic organs namely bone, lymph nodes, subcutaneous tissue, or lung.

The approval comes as welcome news for the company as it unlocks additional funding of $34.9m, from its Private Investment in Public Equity (PIPE) financing tranch. Additionally, Delcath’s stock shot to more than 80% in pre-market trading on 15 August following the approval announcement, compared to the market close on the previous day.

Delcath plans to launch the Hepzato Kit in Q4 2023, with the company’s CEO stating: "We look forward to partnering with cancer centres across the country to build a network of treatment sites trained in the use of this novel therapy."

The Hepzato Kit has been approved in Europe since 2012 and is marketed as Chemosat hepatic delivery system for liver cancers. It uses percutaneous infusion to administer melphalan hydrochloride through Delcath’s proprietary filters for more than 30 minutes followed by a 30-minute washout.

The US approval was based on an open-label Phase III study (NCT02678572). The trial administered the Hepzato Kit for up to six treatments, with a gap of six to eight weeks in between treatments, to both treatment-naïve and previously treated adults with hepatic and extrahepatic lesions. The objective response rate for the treatment was 36.3%, with the response lasting for 14 months (median duration of response).

Approximately 73.6% of the 91 patients enrolled in the trial reported clinical benefit, with 7.7% and 28.6% of the patients reporting complete and partial responses, respectively. The most common side effect of the therapy is a decrease in blood cell counts.

Delcath plans to expand indications for the Hepzato Kit to include cholangiocarcinoma and other primary and metastatic liver cancers.