FDA Expands Approved Use of Stivarga to Treat Liver Cancer

First new liver cancer clearance in nearly a decade

The FDA has expanded the approved use of regorafinib (Stivarga, Bayer HealthCare Pharmaceuticals) to include the treatment of patients with hepatocellular carcinoma (HCC) who have been treated with sorafenib. This is the first FDA-approved treatment for liver cancer in almost a decade.

Regorafinib is a kinase inhibitor that blocks several enzymes involved in promoting cancer growth, including enzymes in the vascular endothelial growth factor pathway. Regorafinib has also been approved to treat colorectal cancer and gastrointestinal stromal tumors that no longer respond to previous treatments.

The safety and efficacy of regorafinib for the treatment of patients with HCC were studied in a randomized trial of 573 patients whose tumors had progressed after receiving sorafenib. The trial measured overall survival, progression-free survival (PFS), and the overall response rate (ORR).

The median overall survival for patients treated with regorafinib was 10.6 months compared with 7.8 months for patients receiving placebo. The median PFS for patients taking regorafinib was 3.1 months compared with 1.5 months for placebo-treated patients. The ORR for patients in the regorafinib group was 11% compared with 4% in the placebo group.

Common adverse effects of regorafinib 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.

Treatment with regorafinib is associated with serious risks, including hepatotoxicity, infections, hemorrhage, gastrointestinal perforation or fistula, dermatological toxicity, hypertension, cardiac ischemia and infarction, reversible posterior leukoencephalopathy syndrome, and wound-healing complications.

Source: FDA; April 27, 2017.