Greater Access to GIST Rx During FDA Review

Gastrointestinal stromal tumors drug submitted for new drug application

(RxWiki News) Gastrointestinal stromal tumors (GIST) are known as soft tissue sarcomas. They can appear anywhere along the digestive tract. They most commonly start in the stomach. A new drug to treat GIST is headed for review by the U.S. Food and Drug Administration (FDA).

Bayer HealthCare and Onyx Pharmaceuticals have submitted a New Drug Application to the FDA for its drug called regorafenib, which is designed to treat advanced GIST.

In clinical trials, the medication was effective in people with GIST that had spread, couldn't be treated with surgery or no longer responded to treatment.

The FDA also allowed the company to provide the drug to patients diagnosed with GIST in qualified settings.

This is part of Bayer’s expanded access program.

"To learn more about clinical trials, visit"

"The submission of regorafenib for the treatment of GIST marks an important milestone for Bayer, bringing us one step closer to potentially addressing a significant medical need for patients with this rare but aggressive disease," said Pamela A. Cyrus, MD, Bayer vice president and head of U.S. medical affairs.

This application is based on findings from a Phase III trial involving 199 patients that showed regaorafenib helped patients live longer before the disease progressed - what's called progression-free survival (PFS).

Study members were given either the best supportive care and regorafenib or best supportive care and a placebo. Those who received regorafenib had 4.8 months of stable disease compared to .9 months among those taking a placebo.

Patients taking a placebo were offered the investigational drug after their disease got worse.

The most common side effects from the drug were hand-foot skin reactions (sores), high blood pressure, diarrhea, fatigue and mouth ulcers.

Findings from the trial were presented at the 2012 American Society of Clinical Oncology meeting in June.

Regorafenib is also being tested in other tumor types. The drug is not currently approved by either the FDA, the European Medicines Agency (EMA) or other health authorities.

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Review Date: 
August 30, 2012