Regorafenib (Stivarga) receives FDA approval for liver cancer treatment

On April 27, 2017, the FDA, for hepatocellular carcinoma patients previously treated with sorafenib, granted approval for regorafenib (brand name Stivarga) to be used.

Liver cancer has few treatment options, especially when advanced, and chemotherapy has limited efficacy. Before this approval, there was no approved second-line treatment for advanced liver cancer after sorafenib.

- Related Topic: Liver cancer doubled in high-income countries Regorafenib previously received approval in 2012 for stage 4 (metastatic) colorectal cancer and, in 2013, advanced Gastrointestinal Stromal Tumors, from the FDA .

Liver cancer received this expanded approval based on an international multicenter randomized double-blind placebo-controlled study in patients with documented progression/worsening after sorafenib, Child Pugh A and stage B or C hepatocellular carcinoma, totaling 573 patients.

During each 28-day cycle, for the first 21 days, patients received oral regorafenib 160 mg once daily and supportive care , or placebo (without active ingredient) plus supportive care, randomized into two groups.

Treatment continued until progression or unacceptable toxicity. Mean overall survival significantly improved to 10.6 months in the regorafenib arm versus 7.8 months in the placebo arm.

What is regorafenib’s mechanism of action?

Regorafenib is involved in normal cell functions, oncogenesis, tumor angiogenesis and the tumor microenvironment formation and related pathological processes as a small-molecule inhibitor of multiple membrane-bound and intracellular kinases.

Safety was assessed in 1142 patients in randomized placebo-controlled studies. Common side effects in at least %20 were pain, hand-foot skin reaction, fatigue, diarrhea, decreased appetite, hypertension, infection, dysphonia, elevated bilirubin, fever, mucositis, weight loss, rash and nausea.

The recommended dose during each 28-day cycle, on the first 21 days, is oral regorafenib after a low-fat meal once daily at 160 mg (four 40 mg tablets).

Stivarga entered reimbursement coverage for liver cancer in June 2018

Coverage applies after progression during or after sorafenib (Nexavar) in locally advanced or metastatic hepatocellular cancer with good liver function (Child-Pugh A), for treatment until progression.

For liver cancer meeting these conditions, Stivarga is prescribed by medical oncologists in one-month doses, with a medical board report including a medical oncologist valid for at most 6 months.