FDA Approves Cabometyx to Treat Aggressive Form of Liver Cancer

The Food and Drug Administration (FDA) approved Cabometyx (cabozantinib) tablets for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar (sorafenib), according to Exelixis, the drug’s manufacturer.

“This new indication for CABOMETYX is an important treatment advance for patients with this aggressive form of liver cancer, a community in need of new therapeutic options,” said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis, said in a press release.

The agency based its approval on results from the pivotal phase 3 CELESTIAL trial — designed to evaluate Cabometyx compared with placebo in 707 patients with advanced HCC, which is the most common form of liver cancer and the fastest-rising cause of cancer-related death in the U.S., according to the release.

In the randomized, double-blind, placebo-controlled study, patients were randomized to receive 60 mg of Cabometyx daily (470 patients) or placebo (237 patients).

Overall survival served as the primary endpoint, while secondary endpoints included objective response rate and progression-free survival — or the time until disease progression or worsening.

Patients treated with Cabometyx demonstrated superior overall survival compared with placebo (10.2 vs. 8 months), and median progression-free survival was more than double (5.2 vs. 1.9 months) compared with placebo.

Four percent of percent of patients in the Cabometyx arm experienced an objective response rate compared with .04 percent of those who received placebo. In addition, disease control (partial response or stable disease) was achieved by 64 percent compared with 33 percent, respectively.

The safety profile of Cabometyx appeared consistent with previous findings. The most common grade 3 or 4 side effects in the treatment arm included hand-foot syndrome (17 percent vs. 0 percent), hypertension (16 percent vs. 2 percent), increased aspartate aminotransferase (12 percent vs. 7 percent), fatigue (10 percent vs. 4 percent) and diarrhea (10 percent vs. 2 percent) compared with placebo. Sixteen percent of patients in the Cabometyx arm and three percent of patients in the placebo arm discontinued treatment due to treatment-related side effects.

“Patients with this form of advanced liver cancer have few treatment options, particularly once their disease progresses following treatment with sorafenib,” lead investigator Ghassan K. Abou-Alfa, M.D., from Memorial Sloan Kettering Cancer Center, said in the release. “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.”