Regorafenib: A Review in Hepatocellular Carcinoma

Area of Science:

• Oncology
• Pharmacology

Background:

• Hepatocellular carcinoma (HCC) is a primary liver cancer with limited treatment options.
• Sorafenib is a standard first-line treatment, but many patients progress.
• There is a need for effective second-line therapies for advanced HCC.

Purpose of the Study:

• To evaluate the efficacy and safety of regorafenib as a second-line treatment for HCC.
• To determine if regorafenib improves overall survival (OS) in patients progressing on sorafenib.

Main Methods:

• The RESORCE trial was a randomized, double-blind, placebo-controlled, multinational, phase III study.
• Patients with advanced HCC who progressed on sorafenib were randomized to receive regorafenib or placebo.
• Overall survival (OS) was the primary endpoint.

Main Results:

• Regorafenib significantly prolonged OS, progression-free survival (PFS), and time to progression (TTP) compared to placebo.
• The OS benefit was primarily attributed to disease stabilization.
• Common adverse events included hand-foot skin reaction, fatigue, diarrhea, and hypertension; no fatal hepatic failure was reported.

Conclusions:

• Regorafenib is the first approved targeted therapy for HCC patients progressing on sorafenib.
• It offers a significant survival benefit and has an acceptable safety profile.
• Regorafenib represents an important new treatment option for this patient population.