The U.S. Food and Drug Administration expanded the approved use of Stivarga (regorafinib) to include treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with the drug sorafenib. This is the first FDA–approved treatment for a liver cancer in almost a decade.

Stivarga is a kinase inhibitor that works by blocking several enzymes that promote cancer growth, including enzymes in the vascular endothelial growth factor pathway. Stivarga is also approved to treat colorectal cancer and gastrointestinal stromal tumors that are no longer responding to previous treatments.

The safety and efficacy of Stivarga for treatment of HCC were studied in a randomized trial of 573 patients with HCC whose tumors had progressed after receiving sorafenib. The trial measured the length of time the patients overall survival, progression–free survival and overall response rate. The median overall survival for patients taking Stivarga was 10.6 months, compared to 7.8 months for patients taking a placebo. The median progression–free survival for patients taking Stivarga was 3.1 months compared to 1.5 months for patients taking a placebo. The overall response rate for patients taking Stivarga was 11 percent, compared to 4 percent of patients taking placebo.

Common side effects of Stivarga include pain (including gastrointestinal and abdominal pain), hand–foot skin reaction, fatigue, diarrhea, decreased appetite, hypertension, infection, dysphonia, hyperbilirubinemia, fever, mucositis, weight loss, rash and nausea. Stivarga is associated with serious risks, hepatotoxicity, infections, hemorrhage, gastrointestinal perforation or fistula, dermatologic toxicity, hypertension, cardiac ischemia and infarction, reversible posterior leukoencephalopathy syndrome and wound healing complications.

Women who are pregnant or breastfeeding should not take Stivarga because it may cause harm to a developing fetus or a newborn baby. Women and men who are taking Stivarga should use effective contraception during and for two months after taking the final dose.