- The U.S. Food and Drug Administration (FDA) has approved cabozantinib for patients with hepatocellular carcinoma (HCC) who were previously treated with sorafenib.
Why this matters
- Cabozantinib inhibits VEGF receptors 1, 2, and 3, and the receptor tyrosine kinases MET and AXL. MET and AXL are associated with poor outcomes and MET expression may be increased following sorafenib exposure.
- The approval was based on results from the CELESTIAL trial:
- 707 patients with HCC who were previously treated with sorafenib and had Child Pugh Class A liver impairment were randomized 2:1 to cabozantinib 60 mg orally once daily or placebo.
- Median OS: 10.2 months versus 8.0 months in placebo (HR, 0.76; P=.0049).
- Median progression free survival was 5.2 months versus 1.9 months for placebo (HR, 0.44; P<.001>
- Objective response rate: 4% versus 0.4%.