FDA Approves Treatment for Gastrointestinal Stromal Tumors

The US Food and Drug Administration (FDA) Monday approved the use of Stivarga (regorafenib) to treat certain advanced gastrointestinal stromal tumors (GISTs). The drug is already approved to treat colon and rectal cancer. The new approval is for GIST that cannot be removed by surgery or has spread to other parts of the body (metastasized) and is no longer responding to other drugs used to treat the disease.

“Stivarga is the third drug approved by the FDA to treat gastrointestinal stromal tumors,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research in a statement. “It provides an important new treatment option for patients with GIST in which other approved drugs are no longer effective.”

GIST can start anywhere in the gastrointestinal tract, but most often occurs in the stomach or small intestine. The disease is not common. An estimated 4,000 to 5,000 new cases are diagnosed each year in the US.

The FDA evaluated Stivarga under its priority review program, which means the FDA tries to make a decision within 6 months instead of the usual 10 months. The drug was also granted orphan product designation, which provides incentives to the drug’s developers because it is intended to treat a rare disease.

Stivarga works by blocking proteins on or near the surface of a cell that help cancer cells grow. By blocking these proteins, Stivarga may help stop the growth of tumors. In a clinical study of 199 patients, those who took Stivarga went almost 4 months longer without the cancer getting worse than patients who took a placebo (dummy pill).

The most common side effects of the drug were weakness and fatigue, pain and redness in the hands and feet, rash, diarrhea, loss of appetite, nausea, weight loss, high blood pressure, mouth sores, infection, voice changes, stomach pain, and fever.

Rare, but serious, side effects included liver damage, severe bleeding, blistering and peeling of skin, very high blood pressure, heart attack, and perforated intestines.

Stivarga is marketed by Bayer HealthCare Pharmaceuticals.

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