Bayer's regorafenib overcomes resistance in patients with rare sarcoma

A new targeted drug - German drug major Bayer’s (BAYN: DE) regorafenib (trade name Stivarga) - demonstrated its ability to control metastatic gastrointestinal stromal tumor, an uncommon and life-threatening form of sarcoma, after the disease had become resistant to all existing therapies, report investigators at Dana-Farber Cancer Institute who led the worldwide clinical trial.

The treatment of gastrointestinal stromal tumor, or GIST, even in its advanced metastatic stage, has been dramatically improved with two oral targeted drugs – imatinib (Gleevec, from Novartis) and sunitinib (Sutent from Pfizer). To date, these have represented the only two Food and Drug Administration-approved treatments with the proven ability to control GIST. However, in more than 85% of patients, GIST becomes resistant to these drugs after seven years and the disease worsens with fatal results.

Bayer submitted a New Drug Application to the US FDA in the summer for its 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 (The Pharma Letter August 31) and is now under accelerated review by the agency. Regorafenib was FDA-approved in September 2012 to treat metastatic colon cancer after failure of standard chemotherapy.