Regorafenib now FDA-approved to treat hepatocellular carcinoma

Approved use of the drug has been expanded to include treatment of patients with hepatocellular carcinoma who have been treated with sorafenib.


The FDA has approved the first treatment for hepatocellular carcinoma in nearly a decade, the agency announced on April 27.

Approved use of regorafenib (Stivarga) has been expanded to include treatment of patients with hepatocellular carcinoma who have been treated with sorafenib, according to an FDA press release. The FDA granted the drug application priority review and designated regorafenib as an orphan drug.

Regorafenib, a kinase inhibitor, works by blocking enzymes that promote cancer growth, including those in the vascular endothelial growth factor pathway. The drug is also approved to treat colorectal cancer and gastrointestinal stromal tumors that have stopped responding to previous treatments.

In a randomized trial of 573 patients with hepatocellular carcinoma whose tumors progressed after initially receiving sorafenib, median overall survival for patients taking regorafenib was 10.6 months, compared to 7.8 months for those who received placebo. Median progression-free survival was 3.1 months in the regorafenib group and 1.5 months in the placebo group. Overall, 11% of patients taking the drug experienced complete or partial shrinkage of their tumors, compared to 4% of those receiving placebo.

Common drug side effects include pain, hand-foot skin reaction, fatigue, diarrhea, decreased appetite, hypertension, infection, dysphonia, hyperbilirubinemia, fever, mucositis, weight loss, rash, and nausea. Serious risks include hepatotoxicity, infections, hemorrhage, gastrointestinal perforation or fistula, dermatologic toxicity, hypertension, cardiac ischemia and infarction, reversible posterior leukoencephalopathy syndrome, and wound healing complications. Regorafenib is not indicated for women who are pregnant or breastfeeding. Individuals taking the drug should use effective contraception during treatment and for two months after taking the final dose.