FDA Approval Likely to Change Initial Treatment for Some People with Advanced Colorectal Cancer
Regulatory approval news: On May 2, 2025, the FDA approved nivolumab plus ipilimumab for first-line treatment of advanced colorectal cancer with MSI-H or dMMR tumors (~5% of advanced cases). The decision was based on updated CheckMate-8HW results showing longer progression-free survival (PFS) versus nivolumab alone (3-year PFS ~68% vs 51%). Serious adverse events and discontinuations were higher with the combination; treatment-related deaths occurred in both arms.
FDA approval: nivolumab + ipilimumab for first-line MSI-H/dMMR advanced colorectal cancer
Type: Regulatory approval
Date reported: May 2, 2025
The US Food and Drug Administration (FDA) has approved nivolumab (Opdivo) plus ipilimumab (Yervoy) for initial (first-line) treatment of advanced colorectal cancer in patients whose tumors are MSI-H or mismatch repair–deficient (dMMR).
The approval was based on updated findings from the phase 3 CheckMate-8HW trial (manufacturer-funded; Bristol Myers Squibb), in which >800 participants with advanced MSI-H/dMMR colorectal cancer were randomized to nivolumab + ipilimumab, nivolumab alone, or chemotherapy (± targeted therapy).
In the updated comparison, the combination produced substantially longer progression-free survival (PFS) than nivolumab alone. At 3 years, approximately 68% of patients in the combination group were alive without progression versus 51% with nivolumab alone.
Safety signals favored monotherapy: serious side effects occurred in 22% with the combination vs 14% with nivolumab alone, and treatment discontinuation due to adverse events was 14% vs 6%. There were two treatment-related deaths in the combination arm and one in the nivolumab-alone arm.
Investigators and commentators highlighted the need for accurate MSI-H/dMMR testing, noting frequent discrepancies between local testing and central laboratory confirmation in the trial.