Pfizer(PFE.US)'s oral anticancer drug Braftovi approved by the FDA for the treatment of colorectal cancer

The U.S. Food and Drug Administration (FDA) officially approved the oral anti-cancer drug Braftovi, developed in collaboration with Pfizer (PFE.US) and Japanese Ono Pharmaceutical, for first-line combination treatment of colorectal cancer on Tuesday. The decision was based on data from Pfizer’s Phase 3 BREAKWATER clinical trial. The FDA permits this once-daily medication to be used as part of combination therapy for previously untreated patients with metastatic colorectal cancer carrying the BRAF V600E mutation.

After the BREAKWATER study met one of its dual primary endpoints related to confirmed overall response rate, the FDA approved Braftovi for the same indication via accelerated approval in December 2024. Regarding the other dual primary endpoint of progression-free survival, the FDA stated that in the study, the combination of Braftovi with the anti-cancer drug cetuximab resulted in a median progression-free survival of 12.8 months in newly diagnosed patients with BRAF V600E-mutant metastatic colorectal cancer.

Braftovi’s active ingredient, encorafenib, is an oral small-molecule BRAF inhibitor. Data show that BRAF mutations occur in up to 15% of metastatic colorectal cancer patients, who generally have a poor prognosis. The BRAF V600E mutation is the most common BRAF mutation, and CRC patients with this mutation have a mortality rate more than twice that of patients with wild-type BRAF.