The U.S. Food and Drug Administration has approved encorafenib (marketed as BRAFTOVI) in combination with cetuximab (marketed as ERBITUX) as a second-line therapy for the treatment of adult patients with metastatic colorectal cancer with a BRAFV600E mutation. 

“BRAF mutations are estimated to occur in up to 15% of people with metastatic colorectal cancer and represent a poor prognosis for these patients,” said Dr. Scott Kopetz, Associate Professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center. “(This) is a much-needed new treatment option for these patients.”

The BRAFV600E mutation is the most common BRAF mutation. The risk of mortality in colorectal cancer patients with the BRAFV600E mutation is more than two times higher than for those with wild-type BRAF, according to Pfizer. 

All patients with metastatic colorectal cancer should be tested for biomarkers, including the BRAF mutation. Patients can learn if they have the BRAFV600E mutation by asking their doctor for test results. Patients who find out they have the BRAFV600E mutation should discuss a treatment plan tailored for their tumor.

BRAFV600E-mutant metastatic CRC has been an area of high unmet need. Encorafenib plus cetuximab is the first and only FDA-approved targeted regimen specifically for this population. Doctors can prescribe this regimen after patients receive prior therapy. 

In the phase III trial that preceded the FDA’s approval, encorafenib plus cetuximab prolonged patients’ survival compared to the control group, which was provided with irinotecan with cetuximab or FOLFIRI with cetuximab. The results of the trial were published in The New England Journal of Medicine. Dr. Kopetz was the lead author of the paper.

“We’re overjoyed to share news of the FDA’s approval of this combination, which will improve outcomes for patients with BRAF-mutant metastatic colorectal cancer,” said Dr. Ronit Yarden, the Alliance’s Senior Director of Medical Affairs. “Personalized medicine such as this is an important pathway toward ending colorectal cancer, and we applaud Pfizer for their innovative work benefitting patients.” 

The most common side effects associated with encorafenib plus cetuximab combination were diarrhea, dermatitis acneiform (acne-like bumps), fatigue, nausea, abdominal and joint pain, anemia, and decreased appetite.