FDA gives accelerated approval to Keytruda for previously treated hepatocellular carcinoma.- Merck Inc.

Merck Inc announced that the FDA has approved Keytruda (pembrolizumab), Merck�s anti-PD-1 therapy, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The approval was based on data from KEYNOTE-224, a single-arm, open-label, multicenter trial evaluating Keytruda in 104 patients with HCC who had disease progression on or after sorafenib or were intolerant to sorafenib.

In KEYNOTE-224, the ORR was 17 percent (95% CI, 11-26), with a complete response rate of 1 percent and a partial response rate of 16 percent. Among the responding patients (n=18), 89 percent experienced a DOR for six months or longer and 56 percent experienced a DOR for 12 months or longer. Among the 104 patients in KEYNOTE-224, the median duration of exposure to Keytruda was 4.2 months (range, 1 day to 1.5 years). Adverse reactions occurring in patients with HCC were generally similar to those 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%). Laboratory abnormalities (Grades 3-4) that occurred at a higher incidence were elevated AST (20%), ALT (9%), and hyperbilirubinemia (10%).