The approved use of Stivarga (regorafenib) has been expanded by the FDA to include treatment of patients with advanced gastrointestinal stromal tumors (GIST) that cannot be surgically removed and no longer respond to other FDA-approved treatments for the disease.
GIST is a tumor in which cancerous cells form in the tissues of the gastrointestinal tract. Stivarga, a multi-kinase inhibitor, blocks several enzymes that promote cancer growth. With this new approval, Stivarga is intended to be used in patients whose GIST cancer cannot be removed by surgery or has spread to other parts of the body (metastatic) and is no longer responding to Gleevec (imatinib) and Sutent (sunitinib), the two other FDA-approved drugs for GIST.
The safety and effectiveness of Stivarga for this use were evaluated in a clinical study of 199 patients with GIST that could not be surgically removed and progressed after treatment with Gleevec or Sutent. Patients were randomly assigned to receive either Stivarga or a placebo. All patients also received optimal supportive care, which includes treatments to help manage side effects and symptoms of cancer.
Patients in the study took Stivarga or placebo until either the cancer progressed or the side effects became unacceptable. Results showed patients who took Stivarga had a delay in tumor growth (progression-free survival) that was, on average, 3.9 months later than patients who were given placebo. Patients who received the placebo were given the opportunity to switch to Stivarga when their cancer progressed.
The most common side effects reported in patients treated with Stivarga were weakness and fatigue, hand-foot syndrome (also called palmar-plantar erythrodysesthesia), diarrhea, loss of appetite, high blood pressure, mouth sores, infection, changes in voice volume or quality, pain, weight loss, stomach pain, rash, fever and nausea.
Serious side effects, which occurred in less than one percent of patients, were liver damage, severe bleeding, blistering and peeling of skin, very high blood pressures requiring emergency treatment, heart attacks and perforations (holes) in the intestines.
Stivarga was approved in September 2012 to treat colorectal cancer. It is marketed by Bayer HealthCare Pharmaceuticals, based in Wayne, N.J. Gleevec is marketed by East Hanover, N.J.-based Novartis, and Sutent is marketed by New York City-based Pfizer.