New Treatment for Liver Cancer Gets FDA Approval

The approval expands the use of the anticancer therapeutic regorafenib (Stivarga) to a third cancer type. It is the first new treatment option for liver cancer in a decade.

The U.S. Food and Drug Administration (FDA) recently expanded the use of regorafenib (Stivarga) to include the treatment of certain patients with the most common form of liver cancer diagnosed in the United States – hepatocellular carcinoma.

Regorafenib is intended for treating patients with hepatocellular carcinoma that has progressed despite treatment with sorafenib (Nexavar).

In 2017, it is expected that there will be 40,710 new cases of liver cancer diagnosed in the United States, according to National Cancer Institute SEER data. While this makes liver cancer the 13th most commonly diagnosed cancer in the country, it is one of the cancer types for which the incidence rate is rising. In fact, it is the cancer with the most rapidly increasing incidence rate among men and women living in the United States. A study published in 2014 in the AACR’s journal Cancer Research projected that liver cancer will become the 11th leading cause of cancer death in 2030.

Sorafenib and regorafenib are antiangiogenic therapeutics that work by impeding the growth of the blood and lymphatic vessel networks that tumors establish to grow and survive. They do this by targeting a combination of the many molecules that promote new blood and lymphatic vessel growth within a tumor. These two therapeutics also target molecules that promote tumor growth and cancer progression in other ways.

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.

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, which were 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.

The FDA approval was rendered on April 27, 2017.