FDA Approves Cabozantinib in Previously Treated Hepatocellular Carcinoma

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On January 14, the U.S. Food and Drug Administration (FDA) approved cabozantinib (Cabometyx) tablets for patients with hepatocellular carcinoma who have been previously treated with sorafenib. The FDA’s approval of this oral tyrosine kinase inhibitor was based on the results of the phase III CELESTIAL trial.

“Patients with this form of advanced liver cancer have few treatment options, particularly once their disease progresses following treatment with sorafenib,” said lead investigator of the CELESTIAL trial, Ghassan K. Abou-Alfa, MD, of Memorial Sloan Kettering Cancer Center. “The results of the -CELESTIAL trial demonstrate that cabozantinib has the efficacy and safety profile to become an important new therapy in our efforts to slow disease progression and improve treatment outcomes.”

More About CELESTIAL Trial

Data from the CELESTIAL trial were originally presented at the 2018 Gastrointestinal Cancers Symposium in January 2018 (Abstract 207)1 and were published in The New England Journal of Medicine2 in July 2018.

In the trial, the median overall survival was 10.2 months with cabozantinib vs 8.0 months with placebo (hazard ratio [HR] = 0.76, 95% confidence interval [CI] = 0.63–0.92; P = .0049). The median progression-free survival was 5.2 months with cabozantinib and 1.9 months with placebo (HR = 0.44, 95% CI = 0.36–0.52; P < .0001). Objective response rates per the Response Evaluation Criteria in Solid Tumors version 1.1 were 4% with cabozantinib and 0.4% with placebo (P = .0086). Disease control (partial response or stable disease) was achieved by 64% of patients in the cabozantinib group compared with 33% of patients in the placebo group.

The most common (≥ 10%) grade 3 or 4 adverse events in the cabozantinib group were palmar-plantar erythrodysesthesia, hypertension, increased aspartate aminotransferase, fatigue, and diarrhea. Treatment-related grade 5 adverse events occurred in six patients in the cabozantinib group and in one patient in the placebo group. Sixteen percent of patients in the cabozantinib arm and 3% of patients in the placebo arm discontinued treatment due to treatment-related adverse events. 


1. Abou-Alfa GK, Meyer T, Cheng A-L, et al: Cabozantinib versus placebo in patients with advanced hepatocellular carcinoma who have received prior sorafenib : Results from the randomized phase III CELESTIAL trial. 2018 Gastrointestinal Cancers Symposium. Abstract 207. Presented January 19, 2018.

2. Abou-Alfa GK, Meyer T, Cheng AL, et al: Cabozantinib in patients with advanced and progressing hepatocellular carcinoma. N Engl J Med 379:54-63, 2018.