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FDA approves Stivarga for patients with advanced GIST
The FDA today expanded the approved use of regorafenib to treat patients with advanced gastrointestinal stromal tumors that cannot be surgically removed and no longer respond to treatment with either imatinib or sunitinib.
The FDA approved regorafenib (Stivarga, Bayer Healthcare) under its priority review program, which allows for accelerated 6-month review of drugs that could offer significant improvement compared with other available treatments or provide an effective therapy where no satisfactory alternative exists.
In addition, the FDA granted regorafenib orphan product designation because it is intended to treat a rare disease.
Richard Pazdur
“Stivarga is the third drug approved by the FDA to treat gastrointestinal stromal tumors,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “It provides an important new treatment option for patients with GIST in which other approved drugs are no longer effective.”
The safety and effectiveness of regorafenib was assessed in a study of 199 patients with GIST that could not be surgically removed and had progressed after treatment with imatinib (Gleevec, Novartis) and sunitinib (Sutent, CPPI CV). Patients were randomly assigned to receive either regorafenib or placebo until disease progression or unacceptable toxicity. All patients also received optimal supportive care, which included treatments to help manage side effects and symptoms of cancer.
Results showed regorafenib delayed tumor growth by an average of 3.9 months longer than placebo. Patients assigned to placebo were given the option to switch to regorafenib when their cancer progressed.
The most common adverse effects observed in patients who received regorafenib included weakness and fatigue, 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.
Perspective
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While great progress has been made in the treatment of GIST since the introduction of kinase inhibitors as effective therapies for this orphan disease, we have been looking for additional effective treatments for GIST patients whose disease worsens despite currently approved therapies. These data show that regorafenib can slow disease progression in patients who are no longer responding to other approved therapies and may provide another avenue for GIST patients who would otherwise have no FDA-approved treatment option.
Perspective
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The demonstration that regorafenib has significant activity in the treatment of GIST refractory to both imatinib and sunitinib represents a significant advance. This observation builds on the already substantial disease control and survival improvement achieved by imatinib in the advanced disease and adjuvant setting. We now have additional clinical proof that alternative, multitargeted tyrosine kinase inhibitors that target cKIT, PDGF and other downstream pathways such as RAF kinase are effective even in refractory GIST. The correlative science from this trial is anxiously awaited, including study of potential pathways of drug resistance and sensitivity. These and other studies will ultimately lead to the more specific use and sequencing of these agents to meet the needs of individual patients. Future drug development in this field is clearly full of great promise, and GIST remains the model solid tumor for the development and clinical implementation of targeted therapies.