The European Medicines Agency (EMA) has recommended approval of retifanlimab (Zynyz) combined with carboplatin and paclitaxel as a first-line treatment for adults with metastatic or inoperable recurrent squamous cell carcinoma of the anal canal.
The recommendation is based on results from the phase 3 POD1UM-303 trial. Patients who received retifanlimab plus chemotherapy had better outcomes than those who received chemotherapy alone. Median progression-free survival increased to 9.3 months compared with 7.4 months, reducing the risk of disease progression or death by 37%. Overall survival improved to 29.2 months versus 23.0 months. The overall response rate was 55.8%, including 22% complete responses, compared with 44.2% in the control group. The median duration of response nearly doubled to 14.0 months versus 7.2 months.
Side effects were common in both groups. Serious (grade 3 or higher) adverse events occurred in 83% of patients receiving retifanlimab and 75% of those receiving chemotherapy alone. Immune-related side effects were more frequent with retifanlimab (49% vs. 26%). This recommendation represents an important advance in a disease area with limited treatment progress in recent decades. Retifanlimab was approved by the U.S. FDA for this use in May 2025.