FDA Expands Approved Use of Stivarga (regorafenib) to Treat Hepatocellular Carcinoma

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

"Limited treatment options are available for patients with liver cancer," said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. "This is the first time patients with HCC have had an FDA-approved treatment that can be used if their cancer has stopped responding to initial treatment with sorafenib."

According to the National Cancer Institute, approximately 40,710 people will be diagnosed with liver cancers in 2017 and approximately 28,920 will die of these diseases. HCC originates in the liver and is the most common form of liver cancer.

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 lived after receiving treatment (overall survival), the length of time tumors did not grow after treatment (progression-free survival) and the percent of patients whose tumors completely or partially shrank after treatment (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, high blood pressure (hypertension), infection, difficulty speaking (dysphonia), high levels of bilirubin in the blood (hyperbilirubinemia), fever, inflammation of the mucous membranes (mucositis), weight loss, rash and nausea. Stivarga is associated with serious risks, including liver damage (hepatotoxicity), infections, heavy bleeding (hemorrhage), holes in the stomach or intestines (gastrointestinal perforation or fistula), skin damage (dermatologic toxicity), hypertension, problems with blood flow to the heart (cardiac ischemia and infarction), temporary brain swelling(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.

This Stivarga application was granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.

This indication for Stivarga also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Stivarga to Bayer HealthCare Pharmaceuticals Inc.