FDA Grants Keytruda Priority Review to Treat Most Common Form of Liver Cancer

The FDA has granted priority review for Keytruda (pembrolizumab) as a treatment for previously treated patients with advanced hepatocellular carcinoma (HCC). The supplemental Biologics Application (sBLA) is also seeking accelerated approval for the new indication.

HCC is the most common type of liver cancer in adults. In the United States, it is estimated that 42,200 new cases of liver cancer will be diagnosed this year, and the incidence has nearly tripled since 1980. HCC has one of the highest mortality rates of solid cancers, with a 5-year survival rate of less than 15%.

The sBLA was submitted based on data from the phase 2 KEYNOTE-224 trial which were recently presented at the 2018 American Society of Clinical Oncology (ASCO) annual meeting. KEYNOTE-224 is a non-randomized, multicenter, open label, phase 2 trial that investigated the efficacy and safety of Keytruda in patients with advanced HCC.

“There continues to be a significant need for new options in the treatment of advanced hepatocellular carcinoma, which is the most common type of liver cancer,” said Scot Ebbinghaus, MD, in a statement. The drug is currently undergoing further assessment in 2 phase 3 randomized trials as a second-line treatment in patients with HCC.

The trial enrolled 104 patients and recorded an objective response in 18 patients (17%; 95% CI:11-26). The best overall responses were 1 patient (1%) that achieved complete response, and 17 (16%) that achieved partial responses. The study saw treatment-related adverse events occur in 76 (73%) of patients, with 16 (15%) being serious.

“The data supporting our application provide a clear rationale for the advancement of the Keytruda clinical program for hepatocellular carcinoma, and we are grateful for the opportunity to work with the FDA to potentially bring Keytruda to patients living with this difficult-to-treat cancer.”

Merck, the developer of Keytruda, is investigating the use of the drug in the treatment of gastrointestinal cancers in more than 10 different trials. The FDA has set a target action date of November 9, 2018.