FDA Approves Regorafenib for Second-line in Hepatocellular Carcinoma

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.

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.

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.

Reference

1. FDA expands approved use of Stivarga to treat liver cancer [news release]. Silver Spring, MD: US Food and Drug Administration; April 27, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555608.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 27, 2017.