Regorafenib, a multikinase inhibitor, was approved by the US Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC) after prior sorafenib therapy.1

Regorafenib is also approved for the treatment of relapsed/refractory colorectal cancer and gastrointestinal stromal tumors. Priority Review designation was granted to regorafenib for this indication, and it was assigned an Orphan Drug designation.

The approval was based on a randomized controlled trial of 573 patients with HCC who had progressed after sorafenib.

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Regorafenib increased median overall survival to 10.6 months from 7.8 months with placebo. The median progression-free survival was also prolonged with regorafenib (3.1 months) compared with placebo (1.5 months).

The response rate was 11% with regorafenib and 4% with placebo.

RELATED: Clinical Pathways: A Cost-effective System for Determining mCRC Treatment

The adverse events associated with regorafenib are similar to other multikinase inhibitors and include gastrointestinal and abdominal pain, hand-foot skin reaction, fatigue, diarrhea, and hypertension.


  1. FDA expands approved use of Stivarga to treat liver cancer [news release]. Silver Spring, MD: US Food and Drug Administration; April 27, 2017. Accessed April 27, 2017.