FDA Approval Summary: Retifanlimab for the Treatment of Adult Patients with Metastatic or Inoperable Locally Recurrent Squamous Cell Carcinoma of the Anal Canal.
Shruti U Gandhy, Sandra J Casak, May Tun Saung et al.
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In 30 seconds
This FDA approval summary describes evidence from POD1UM-303, a randomised trial in 308 patients with advanced squamous cell carcinoma of the anal canal (SCAC) and no prior systemic treatment for advanced disease, and POD1UM-202, a single-arm second-line study. In POD1UM-303, adding retifanlimab to carboplatin and paclitaxel improved median progression-free survival (PFS) versus placebo plus chemotherapy: 9.3 months (95% CI, 7.5-11.3) versus 7.4 months (95% CI, 7.1-7.7; HR, 0.63; P=0.0006). Interim overall survival was not statistically significant. Single-agent retifanlimab had a 14% response rate in POD1UM-202.
Key findings
- In POD1UM-303, median PFS was 9.3 months with retifanlimab plus chemotherapy versus 7.4 months with placebo plus chemotherapy.
- The PFS hazard ratio was 0.63 (95% CI, 0.47-0.84; P value 0.0006).
- Interim overall survival was not statistically significant (HR, 0.70; 95% CI, 0.49-1.01).
- In the single-arm POD1UM-202 study, single-agent retifanlimab produced an independently assessed overall response rate of 14% (95% CI, 8-23).
Why it matters
Advanced SCAC has limited treatment options, and the reported PFS improvement supports retifanlimab’s role in the first-line combination regimen. The second-line response findings provide evidence for its use as a single agent, although the single-arm design limits comparison with other treatments. The absence of new safety signals is also relevant when interpreting the approval evidence.
What to keep in mind
Interim overall survival results were not statistically significant. The second-line evidence came from a single-arm trial, so it does not provide a randomised comparison.