FDA Approves Pembrolizumab to Treat HCC

Earlier this month, Merck announced that the FDA approved its anti—PD-1 therapy, pembrolizumab (sold as Keytruda), for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.

This new indication was approved under the accelerated approval pathway due to tumor response rate and durability of response as demonstrated in the KEYNOTE-224 trial. However, continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.

“Hepatocellular carcinoma is the most common type of liver cancer in adult, and while we have seen recent therapeutic advancements, there are still limited treatment options for advanced recurrent disease,” Andrew Zhu, MD, PhD, lead investigator and director of liver cancer research at Massachusetts General Hospital and professor of medicine at Harvard Medical School, said in a statement.

KEYNOTE-224 is a single-arm, open-label, multicenter study evaluating pembrolizumab in 104 patients with HCC who had disease progression during or after treatment with sorafenib, or who were intolerant to sorafenib. Patients received pembrolizumab 200 mg every 3 weeks until unacceptable toxicity or confirmed disease progression.

The trial found that objective response rate was 17% (95% CI, 11-26), with a complete response rate of 1%, and a partial response rate of 16%. Among the patients who responded to treatment (n = 18), 89% experienced a duration of response for 6 months or longer, and 56% experienced a duration of response for 12 months or longer.

Adverse events occurring in patients with HCC were similar to those seen in patients with melanoma or non-small cell lung cancer, with the exception of increased incidences of ascites (8% grades 3-4), and immune-mediated hepatitis (2.9%).

“We look forward to continuing to advance research for hepatocellular carcinoma across our portfolio with the goal to help even more patients affected by this type of cancer,” said Scot Ebbinghaus, MD, vice president of clinical research at Merck Research Laboratories.