Bayer HealthCare and Onyx Pharmaceuticals, a global biopharmaceutical company based in San Francisco, have submitted a New Drug Application (NDA) to the FDA for the oral multi-kinase inhibitor regorafenib for the treatment of metastatic and/or unresectable gastrointestinal stromal tumors (GIST) in patients whose disease has progressed despite prior treatment.

Regorafenib was developed by Bayer. In 2011, Bayer entered into an agreement with Onyx Pharmaceuticals under which Onyx will receive a royalty on any future global net sales of regorafenib in oncology. Bayer and Onyx will jointly promote regorafenib in the U.S.

"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, vice president and head of U.S. medical affairs, Bayer HealthCare Pharmaceuticals. "With the development of regorafenib and other oncology compounds, we remain committed to discovering and advancing cancer therapies for patients who are in need of new treatment options."

The submission is based on data from the pivotal phase III GRID (GIST – Regorafenib In Progressive Disease) trial, which showed that regorafenib plus best supportive care (BSC) significantly improved progression-free survival (PFS) compared to placebo plus BSC [HR=0.27 (95% CI 0.19-0.39), p

In this study, the most frequent drug-related adverse event were hand-foot skin reaction, hypertension, diarrhea, fatigue and oral mucositis. 

In addition, the FDA recently agreed that Bayer can proceed with its expanded access program (EAP) to provide regorafenib to patients diagnosed with GIST through qualified clinical sites participating in the EAP.