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FDA Grants Orphan Drug Designation to Bayer's Investigational Compound Regorafenib for the Treatment of Gastrointestinal Stromal Tumors

WAYNE, N.J., Feb. 4, 2011 /PRNewswire/ -- Bayer HealthCare Pharmaceuticals Inc. announced today that its investigational compound regorafenib (BAY 73-4506) has been granted orphan drug status for the treatment of patients with gastrointestinal stromal tumors (GIST) by the U.S. Food and Drug Administration (FDA).

Regorafenib is an investigational agent and is not approved by the FDA, the European Medicines Agency (EMA) or other Health Authorities.

"This is an important step in the overall development process for this investigational compound," said Kemal Malik, MD, Head of Global Development and member of the Bayer HealthCare Executive Committee.

In January, Bayer began enrolling patients in a randomized, double-blind, placebo-controlled Phase III study of regorafenib plus best supportive care versus placebo plus best supportive care for subjects with metastatic and/or unresectable gastrointestinal stromal tumors (GIST) whose disease has progressed despite prior treatment with at least imatinib and sunitinib (NCT01271712). The trial is estimated to enroll 170 patients, who will be randomized in a 2:1 ratio to receive either regorafenib or placebo. Subjects receiving placebo who experience disease progression may be offered open-label regorafenib treatment (cross-over option).

The primary endpoint of this trial is progression-free survival (PFS), and secondary endpoints include overall survival (OS), time to progression (TTP), disease control rate (DCR), tumor response rate (RR), duration of response (DOR), and safety. All patients will enter the Survival Follow-Up Period upon discontinuation of study treatment, during which assessment of survival status will be performed every three months.

For information about this study, please visit (NCT01271712).

Orphan Drug Designation in the United States

In the United States, the Orphan Drugs Act (ODA) provides for the orphan drug designation which aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not shorten the duration of the regulatory review and approval process.  

About GIST

GIST is the most common form of sarcoma involving the gastrointestinal tract. GISTs arise in the GI tract, with most primary tumors originating in the stomach or small intestine. GIST represents a potentially life-threatening malignancy if the disease has spread to other parts of the body (metastasized) or is unable to be surgically removed with curative intent. The incidence of GIST is estimated to be 11 to 20 patients per million per year. The international prevalence of GISTs is comparable to that reported in the United States. In the U.S., it is estimated that there are approximately 4,500-6,000 new cases of GISTs diagnosed each year, of which about 1,500 have already metastasized when they are initially found. GISTs are difficult to diagnose and are usually found incidentally when the doctor is looking for other problems.

About Bayer HealthCare Pharmaceuticals Inc.

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions.  As a specialty pharmaceutical company, Bayer HealthCare Pharmaceuticals provides products for Diagnostic Imaging, General Medicine, Hematology, Neurology, Oncology and Women's Healthcare.  The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at  The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

SOURCE Bayer HealthCare Pharmaceuticals Inc.
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