FDA Expands Approved Use of Regorafenib for Hepatocellular Carcinoma


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

“Limited treatment options are available for patients with liver cancer,” said Richard Pazdur, MD, 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. Hepatocellular carcinoma originates in the liver and is the most common form of liver cancer.

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


The safety and efficacy of regorafenib for treatment of hepatocellular carcinoma were studied in the randomized phase III RESORCE trial of 573 patients with the disease whose tumors had progressed after receiving sorafenib.

Median overall survival for patients randomized to treatment with regoraenib was 10.6 months, compared to 7.8 months for patients taking a placebo. The median progression-free survival for patients taking regorafenib was 3.1 months, compared to 1.5 months for patients taking a placebo. The overall response rate for patients taking regorafenib was 11%, compared to 4% of patients taking placebo.

Common side effects of regorafenib 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. Regorafenib is also associated with serious risks, including hepatotoxicity, infections, hemorrhage, gastrointestinal perforation or fistula, dermatologic toxicity, hypertension, cardiac ischemia and infarction, reversible posterior leukoencephalopathy syndrome, and wound-healing complications.

The recommended regorafenib dose is 160 mg (four 40-mg tablets) taken orally once daily after a low-fat meal for the first 21 days of each 28-day cycle.

Full prescribing information is available at

This regorafenib application was granted Priority Review designation, under which the FDA’s goal is to take action on an application within 6 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 regorafenib also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.