The US FDA has awarded Breakthrough Therapy Designation to encorafenib (braftoviTM) combined with binimetinib (mektovi®) and cetuximab for use as an investigational (not FDA-approved) treatment of BRAFV600E–mutant metastatic colorectal cancer. The experimental treatment is designed for cancer that has been identified using a specific type of test, and the treatment is intended for patients who have had a failure of one or two lines of therapy. You can find more detailed information in the source press release here, at PR Newswire.
About Colorectal Cancer
Colorectal cancer, also known as bowel cancer, originates in the colon or rectum. According to the source press release, an estimated 1.4 million people were diagnosed with this form of cancer in 2012, and it is the second most common form of cancer in women worldwide, and third most common in men.
The investigational drug combination has been granted Breakthrough Therapy designation to treat patients with BRAFV600E–mutant metastatic colorectal cancer. Metastatic cancer is cancer that spreads to another area of the body. BRAF alterations, according to the source press release, are thought to occur in approximately 10% – 15% of people diagnosed with colorectal cancer. They are associated with poor prognoses, and patients with colorectal cancer with the BRAFV600E alteration have over two times the risk of mortality compared to patients who have wild-type BRAF.
About Breakthrough Therapy Designation
The US FDA’s Breakthrough Therapy designation is intended to speed up the development and review process for drugs that are designed as a potential treatment for a serious condition, and where initial evidence suggests that the drug may provide a significant improvement in treatment options to patients.
Research into the Investigational Combination Therapy
Results from the on-going Phase 3 clinical trial called Beacon CRC were shared in June this year. Beacon CRC is an open-label study exploring the effectiveness and safety of a combination of encorafenib, binimetinib, and cetuximab for patients who have BRAFV600E–mutant metastatic colorectal cancer, amongst other eligibility criteria.
The announced results included an overall response rate of 48%, and 62% for patients who had only received one prior line of therapy. In addition, the drug combination was considered to be “generally well-tolerated”. The most common adverse event was anaemia, which affected 13% of patients.