FDA greenlights retifanlimab combo and monotherapy for advanced anal cancer: a new era in anal squamous cell carcinoma treatment

The occurrence of anal squamous cell carcinoma (ASCC), predominantly driven by persistent HPV infection, is increasing all over the world. The phase-3 POD1UM trial assessed adding the PD-1 inhibitor retifanlemab to standard Carboplatin-paclitaxel chemotherapy in 308 previously untreated patients with advanced ASCC. The combination displayed greater potency, markedly improving median overall survival (29.2 vs 23 months) and clinical response rate (55.8% vs 44.2%), with 22% achieving complete response. Benefits were persistent across most subgroups, though not in PD-L1 negative or HIV positive patients. As a consequence, the FDA has approved retifanlimab both as monotherapy and in combination, marking a major development that furnish new hope and upgrade survival outcomes for patients with this disorder.