NDA Submitted for Regorafenib for Gastrointestinal Stromal Tumors (GIST)

Bayer HealthCare and Onyx announced that Bayer HealthCare has submitted a New Drug Application (NDA) to the FDA for regorafenib for the treatment of metastatic and/or unresectable gastrointestinal stromal tumors (GIST) in patients whose disease has progressed despite prior treatment.

The submission is based on data from the pivotal Phase 3 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<0.0001] in patients with metastatic and/or unresectable GIST who were previously treated with imatinib (Gleevec; Novartis) and sunitinib (Sutent; Pfizer). The median PFS was 4.8 months in the regorafenib arm vs. 0.9 months in the placebo arm and there was a positive trend in the regorafenib group in improving overall survival (OS) [HR=0.77 (95% CI 0.42–1.41), P=0.2]. In addition, the study design allowed patients receiving placebo to cross-over following disease progression.

Regorafenib is an investigational oral multi-kinase inhibitor currently being studied in clinical trials for its potential to treat patients with various tumor types.

For more information visit www.onyx.com or www.bayer.com.