FDA Approves First Liver Cancer Treatment in a Decade

On Thursday, the U.S. Food and Drug Administration (FDA) expanded the use of the antiangiogenic therapeutic regorafenib (Stivarga) to include the treatment of certain patients with liver cancer. Specifically, regorafenib is intended for treating patients with hepatocellular carcinoma—the most common form of liver cancer diagnosed in U.S. adults—that has progressed despite treatment with another antiangiogenic therapeutic, sorafenib (Nexavar).

Antiangiogenic therapeutics target a combination of the many molecules that promote the growth of the blood and lymphatic vessel networks that tumors establish to grow and survive. In many cases, these therapeutics also target molecules that promote tumor growth and cancer progression in other ways.

Regorafenib impedes the growth of the blood vessel networks that tumors establish in order to grow and survive.

Because each antiangiogenic therapeutic targets a slightly different combination of molecules, if a patient’s tumor develops resistance to one antiangiogenic therapeutic, he or she is often treated with another member of this expansive class of anticancer therapeutics.

The antiangiogenic therapeutic sorafenib was approved by the FDA for treating advanced hepatocellular carcinoma in 2007. Since then, no new treatments have been approved for patients with this disease, and the overall five-year relative survival rate for patients with hepatocellular carcinoma is around 12 percent.

The approval of regorafenib for advanced hepatocellular carcinoma was based on results from the randomized, phase III RESORCE clinical trial. Initial results from the trial, published recently in The Lancet, showed that regorafenib improved overall survival for patients with hepatocellular carcinoma that had progressed despite treatment with sorafenib compared with placebo. Specifically, median overall survival was 10.6 months for the regorafenib arm compared with 7.8 months for the placebo arm. The FDA announcement also stated that 11 percent of patients had a response following regorafenib treatment.

This decision by the FDA expands the number of cancer types for which regorafenib is approved. As discussed in the AACR Cancer Progress Report 2013, it was approved for treating certain patients with colorectal cancer and certain patients with gastrointestinal stromal tumors in 2012 and 2013, respectively.