Cabometyx filed with FDA for treating previously treated advanced hepatocellular carcinoma.- Exelixis.

Exelixis has completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Cabometyx (cabozantinib) tablets as a treatment for patients with previously treated advanced hepatocellular carcinoma (HCC). The sNDA submission is based on results from the CELESTIAL randomized pivotal phase III trial of Cabometyx in patients with advanced HCC who received prior sorafenib. CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries.

The trial was designed to enroll 760 patients with advanced HCC who received prior sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms during the blinded treatment phase of the trial. The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.