Keytruda Wins FDA Approval for Hepatocellular Carcinoma


The FDA approved Merck’s pembrolizumab (Keytruda) for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib.

The FDA approved Merck’s pembrolizumab (Keytruda) for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib, according to a press release.

The latest approval, which was granted under accelerated approval based on tumor response rate and durability of response, marks the 14th indication for pembrolizumab, Merck announced.

In the KEYNOTE-224 phase 3 clinical trial, pembrolizumab was evaluated in 104 patients with HCC who had disease progression on or after sorafenib or were intolerant to sorafenib. Eligible patients included those with measurable disease and Child-Pugh class A liver impairment, as well as those with active and inactive hepatitis B virus and past or ongoing hepatitis C virus.

For the study, patients received pembrolizumab 200 mg every 3 weeks until unacceptable toxicity or confirmed disease progression, and patients without disease progression were treated for up to 24 months. Tumor status was assessed every 9 weeks.

According to the data, the objective response rate was 17% with a complete response rate of 1% and a partial response rate of 16%. Among the responding patients, 89% experienced a duration of response for 6 months or longer and 56% experienced a duration of response for 12 months or longer.

The median duration of exposure among the patients in the trial was 4.2 months. Common adverse effects reported in those with HCC were generally similar with those in patients with melanoma or non-small cell lung cancer, with the exception of increased incidences of ascites and immune-mediated hepatitis.

“Hepatocellular carcinoma is the most common type of liver cancer in adults, and while we have seen in recent therapeutic advancements, there are still limited treatment options for advanced recurrent disease,” lead investigator Andrew X. Zhu, MD, PhD, director of liver cancer research at Massachusetts General Hospital and professor of medicine at Harvard Medical School, said in a statement. “Today’s approval of Keytruda is important, as it provides a new treatment option for patients with hepatocellular carcinoma who have been previously treated with sorafenib.”

According to Merck, immune-mediated adverse reactions can occur with pembrolizumab, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, severe skin reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation. Based on the severity of the adverse reaction, Merck noted that the treatment should be withheld or discontinued and corticosteroids administered if appropriate.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials, according to the press release.


FDA Approves Merck’s Keytruda (pembrolizumab) for the Treatment of Patients with Hepatocellular Carcinoma (HCC) Who Have Been Previously Treated with Sorafenib [news release]. Merck’s website. Accessed November 9, 2018.

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