The FDA granted breakthrough therapy designation to pembrolizumab in combination with lenvatinib as first-line treatment for patients with advanced unresectable hepatocellular carcinoma who are not able to receive locoregional treatment.
This is the third breakthrough therapy designation granted by FDA for pembrolizumab (Keytruda, Merck) — an anti-PD-1 therapy — and lenvatinib (Lenvima, Eisai), a multiple receptor tyrosine kinase inhibitor.
“As part of our ongoing collaboration with Eisai, we are committed to evaluating the potential of Keytruda plus Lenvima across a number of different types of cancer,” Jonathan Cheng, MD, vice president for oncology clinical research at Merck Research Laboratories, said in a press release. “With this breakthrough therapy designation from the FDA, we look forward to working with Eisai to potentially build upon our existing indications for this difficult-to-treat cancer, so that we can help patients through a combination approach.”
The FDA previously granted breakthrough therapy designation to the pembrolizumab-lenvatinib combination for treatment of advanced or metastatic renal cell carcinoma, and treatment of certain patients with advanced or metastatic endometrial carcinoma.
The agency based the HCC breakthrough therapy designation on interim results from the phase 1B KEYNOTE-524/Study 116, presented at this year’s American Association for Cancer Research Annual Meeting.
The multicenter, open-label, single-arm study included patients with unresectable HCC who had Barcelona Clinic Liver Cancer stage B or C disease, Child-Pugh class A disease, and ECOG performance status of 0 or 1.
Researchers assessed the efficacy and safety of pembrolizumab — dosed at 200 mg via IV every 3 weeks — and lenvatinib, dosed at 12 mg daily for patients who weighed 60 kg or more, or 8 mg per day for patients who weighed less than 60 kg.
Safety and tolerability served as primary endpoints. OS, PFS, objective response rate and time to progression served as secondary endpoints.
“We are excited that the FDA has recognized the potential of Keytruda plus Lenvima in combination in advanced unresectable hepatocellular carcinoma not amenable to locoregional treatment,” Takashi Owa, PhD, vice president and chief medicine creation/chief discovery officer for the oncology business group at Eisai, said in the release. “We are dedicated to working together with Merck to potentially bring another important option to patients.”