The U.S. FDA has approved Delaware-based Delcath's drug/device combo product, branded Hepzato Kit, as a liver-directed treatment for adult patients with metastatic uveal melanoma (mUM), a rare and aggressive form of cancer.
Hepzato Kit is a combination product that administers Hepzato (melphalan), a well-known and long-approved chemotherapeutic agent, directly to the liver through Delcath's novel device delivery system, the Hepatic Delivery System (HDS), which permits higher drug exposure in target tissues while limiting systemic toxicity.
The agency's green light makes the product the only liver-directed therapy approved by the FDA for the treatment of mUM. About 90% of mUM involves the liver, and liver failure is often the cause of death.
The approval has been a long time coming for Delcath, who was hit with a CRL for its original application for the product, then called Melblez Kit, back in 2013. At the time, the FDA said that Delcath must perform another "well-controlled randomized trial(s) to establish the safety and efficacy of Melblez Kit using overall survival as the primary efficacy outcome measure," and which "demonstrates that the clinical benefits of Melblez Kit outweigh its risks."
Delcath followed through with the request and the recent approval was based primarily on the results of the FOCUS study, a phase 3, single arm, multicenter, open label study, which administered Hepzato via the hepatic delivery system during a percutaneous hepatic perfusion procedure. Treatment with Hepzato Kit resulted in an overall response rate of 36.3% including 7.7% of patients with a complete response.
The combo kit comes with a boxed warning, which includes toxicity related to the procedure, myelosuppression and a REMS program to manage and mitigate risks.