The FDA expanded the approved use of 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.
"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 NCI, 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.
Regorafinib is a kinase inhibitor that is also approved to treat colorectal cancer and gastrointestinal stromal tumors that are no longer responding to previous treatments.
The safety and efficacy of regorafinib 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 overall survival, progression-free survival, and overall response rate. The median overall survival for patients taking regorafinib was 10.6 months, compared to 7.8 months for patients taking a placebo. The median progression-free survival for patients taking regorafinib was 3.1 months compared to 1.5 months for patients taking a placebo. The overall response rate was 11 percent, compared to 4 percent of patients taking placebo.
Common side effects of 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. Regorafinib is 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.
Women who are pregnant or breastfeeding should not take regorafinib because it may cause harm to a developing fetus or a newborn baby. Women and men who are taking regorafinib should use effective contraception during and for 2 months after taking the final dose.
This regorafinib application was granted Priority Review designation. This indication also received Orphan Drug designation.