FDA expands approval of Stivarga to include liver cancer

The FDA expanded the approval of regorafenib to include the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib.

Regorafenib (Stivarga, Bayer) — a kinase inhibitor that works by blocking several enzymes that promote cancer growth — is the first FDA–approved treatment for liver cancer in nearly a decade.

A randomized trial compared the safety and efficacy of regorafenib vs. placebo in 573 patients with HCC whose tumors had progressed after receiving sorafenib (Nexavar, Bayer).

Patients treated with regorafenib demonstrated a superior median OS (10.6 vs. 7.8 months), PFS (3.1 vs. 1.5 months) and overall response rate (11% vs. 4%) compared with patients treated with placebo.

“Limited treatment options are available for patients with liver cancer,” 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, said in a press 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.”

Common adverse events include pain, hand–foot skin reaction, fatigue, diarrhea, decreased appetite, high blood pressure, infection, difficulty speaking, high levels of bilirubin in the blood, fever, inflammation of the mucous membranes, weight loss, rash and nausea.

The FDA warned regorafenib is associated with serious risks, including liver damage, infections, heavy bleeding, holes in the stomach or intestines, skin damage, hypertension, problems with blood flow to the heart, temporary brain swelling and wound healing complications.

This application had been granted priority review and orphan drug designation. Regorafenib also is indicated for colorectal cancer and gastrointestinal stromal tumors that are no longer responding to previous treatments.