Bayer HealthCare's regorafenib receives orphan drug status for GIST treatment

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.