Regorafinib Granted Expanded FDA Approval for Liver Cancer

The FDA has expanded the use of regorafinib (Stivarga) to include the treatment of hepatocellular carcinoma (HCC) in patients who have been previously treated with sorafenib.

Regorafinib is a kinase inhibitor designed to block several enzymes that promote cancer growth, including those in the endothelial growth factor pathway. This is the first FDA-approved treatment for liver cancer in nearly a decade, according to a press release.

“Limited treatment options are available for patients with liver cancer,” Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products, FDA’s Center for Drug Evaluation and Research, said in a release. “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.”

The approval was based on a safety and efficacy randomized trial that studies regorafinib in 573 patients with HCC whose tumors had progressed after treatment with sorafenib. The trial measured the overall survival (OS), progression-free survival (PFS), and overall response rate (ORR).

The results of the study showed that the median OS for patients administered regorafinib was 10.6 months compared with 7.8 months in patients who received a placebo. The median PFS for regorafinib was 3.1 months compared with 1.5 months with the placebo, and the ORR was 11% versus 4%, respectively.

Common adverse events (AEs) included, pain, hand-foot skin reaction, fatigue, diarrhea, decreased appetite, hypertension, infection, dysphonia, hyperbilirubinemia, fever, mucositis, weight loss, rash and nausea.

Serious AEs included hepatotoxicity, hemorrhage, gastrointestinal perforation or fistula, dermatologic toxicity, hypertension, cardiac ischemia and infarction, reversible posterior leukoencephalopathy syndrome, and wound healing complications.