The U.S. Food and Drug Administration (FDA) approved the use of Stivarga (regorafinib) for the treatment of patients with hepatocellular carcinoma (HCC), a form of liver cancer, who progressed after receiving Nexavar (sorafenib). This is the first FDA-approved drug for liver cancer in nearly a decade.
Stirvaga already was approved in the U.S. for the treatment of colorectal cancer and gastrointestinal stromal tumors.
“Limited treatment options are available for patients with liver cancer,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a press release by the agency. “This is the first time patients with HCC have had an FDA-approved treatment that can be used if their cancer has stopped responding to initial treatment with sorafenib.”
Stivarga inhibits several enzymes that participate in cancer growth, including enzymes in the vascular endothelial growth factor pathway, which drive the growth of new blood vessels in tumors.
The safety and effectivness of this drug in HCC was investigated in the Phase 3 RESORCE study (NCT01774344), which included 573 patients who did not respond to treatment with Nexavar.
Results showed that the 11% of patients on Stivarga responded to treatment, compared to 4% in the placebo group.
Patients treated with Stivarga lived longer compared to those receiving a placebo (median overall survival of 10.6 months versus 7.8 months). Treated patients also had a larger interval without disease progression compared to the placebo group (median progression-free survival of 3.1 months versus 1.5 months).
The most common side effects associated with this treatment include pain (such as gastrointestinal and abdominal pain), skin reactions in the hands and feet, fatigue, diarrhea, decreased appetite, high blood pressure, infection, difficulty speaking, high levels of bilirubin in the blood, fever, inflammation of the mucous membranes, weight loss, rash, and nausea.
Stivarga also may induce serious adverse side effects, such as liver damage, infections, heavy bleeding, stomach or intestine damage (holes), skin damage, hypertension, heart failure, temporary brain swelling, and wound-healing anomalies, according to the FDA announcement.
Pregnant or breastfeeding women should not take Stivarga, as this treatment may harm the fetus or the newborn baby. The FDA also recommends that people taking this medication use contraception while on treatment and for two months after the last dose.
Hepatocellular carcinoma is the most common type of liver cancer and a leading cause of cancer deaths worldwide. The National Cancer Institute estimates that about 40,710 people will be diagnosed with liver cancer in 2017.
Without treatment, patients have very poor prognosis, usually surviving less than six months after diagnosis.