Cabometyx Gets FDA Approval for Hepatocellular Carcinoma

The Food and Drug Administration has approved Cabometyx (cabozantinib; Exelixis) for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.

The approval of the HCC indication was based on data from the Phase 3 CELESTIAL trial (N=707) which randomized patients who previously received sorafenib to either Cabometyx or placebo. Results showed that treatment with Cabometyx led to a statistically significant and clinically meaningful improvement in overall survival compared with placebo (10.2 months vs 8 months; hazard ratio [HR] 0.76, 95% CI, 0.63–0.92; P =.0049). In addition, median progression free survival was 5.2 months in the Cabometyx group vs 1.9 months in the placebo arm (HR 0.44, 95% CI, 0.36–0.52; P <.0001). The most common adverse events reported in the trial included palmar-plantar erythrodysesthesia, hypertension, increased aspartate aminotransferase, fatigue, and diarrhea.

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

Cabometyx, a kinase inhibitor, is also approved for the treatment of patients with advanced renal cell carcinoma. The product is available in 20mg, 40mg, and 60mg tablets.

For more information visit Exelixis.com.