Regorafenib FAQ

On October 26, 2011, Bayer Healthcare announced positive results from its Phase III trial of regorafenib (BAY 73-4506).

On September 27, 2012, Stivarga (reforafenib) was approved by the FDA.

Dr. Laura Porter, the CCA’s Patient Advocate Medical Consultant and a stage IV colorectal cancer survivor, has summarized what you need to know about this drug and what this news means for the colorectal cancer community.

What is regorafenib? 

Regorafenib is a cancer-fighting drug developed by Bayer Healthcare. It’s an investigational oral multi-kinase inhibitor, which means that it can be taken in pill form and works to block the production of certain proteins necessary for cancer growth. It is designed to treat patients with metastatic colorectal cancer whose cancer has continued to advance after approved standard therapies.

Why were clinical trials for regorafenib stopped?

You have probably heard that the clinical trial for regorafenib was stopped early. This is because a scheduled interim evaluation by an independent committee found statistically significant improvement in overall survival met in the Phase III trial. The trial was a multi-center, international, double-blind randomized (neither the researchers nor the patients knew what they were given), placebo-controlled trial conducted in North America, Japan, China, Europe and Australia.  Once the trial was stopped, those on placebo were able to get the drug. The trial enrolled 760 people with metastatic colorectal cancer that had progressed on standard therapy. The actual figures from the trial will most likely be released early next year.

Why is this new drug exciting news for the colorectal cancer community?

It’s been nearly five years since the FDA has approved a drug for the treatment of colorectal cancer. Regorafenib is an oral inhibitor of multiple tyrosine kinases (TKs). Tyrosine kinases are responsible for “turning on” tumor cells.  By blocking these TKs, regorafenib can stop the growth of the cancer. Currently, there are only three biologics approved by the FDA that target TKs, all of which are administered by IV and cannot be given in combination. These are Avastin, Erbitux and Vectbix. Regorafenib targets the same TKs as these biologics and at least one other TK responsible for tumor proliferation and growth. Regorafenib will allow doctors to combine multiple targets in one oral medication, which will be beneficial to patients on several levels.

When will regorafenib be available to patients?

On September 27, 2012, Stivarga (reforafenib) was approved by the FDA. The CCA will work to keep you updated as this new drug is available to patients.

Would the availability of regorafenib affect the current drug shortage?

Regorafenib would not replace any of the current drugs on the shortage list. In fact, in the trial the regorafenib was given with “Best Supportive Care” as a single agent, which means it was given without the addition of other drugs such as FOLFOX, resulting in fewer drugs and less drug interactions.

"Patients with metastatic colorectal cancer are in need of new treatment options, and these data demonstrate that regorafenib increased overall survival," said Ted W. Love, M.D., Executive Vice President, Research and Development and Technical Operations at Onyx Pharmaceuticals. "We look forward to seeing this promising therapy become available to patients as quickly as possible."

Please continue to visit the Colon Cancer Alliance website for the latest updates on regorafenib and other colorectal cancer news.