Immuno-Oncology Resource Center

Immuno-Oncology Resource Center


Press Release

July 26, 2021
2 min read

FDA issues complete response letter for retifanlimab for anal cancer


Press Release

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

The FDA issued a complete response letter to Incyte indicating it cannot approve the company’s biologics license application that seeks approval of retifanlimab for certain patients with anal cancer.

The agency determined additional data are needed to demonstrate the clinical benefit of retifanlimab (Incyte), a PD-1 inhibitor administered via IV.

Incyte officials will discuss next steps with the FDA, according to a company-issued press release.

Incyte sought approval of retifanlimab for treatment of patients with locally advanced or metastatic squamous cell carcinoma of the anal canal who progressed on or who were intolerant of platinum-based chemotherapy.

As Healio previously reported, the FDA’s Oncologic Drugs Advisory Committee last month voted 13 to 4 in favor of deferring a regulatory decision on retifanlimab for this indication due, in part, to concerns about the low objective response rate reported in the open-label, phase 2 PODIUM-202 trial.

The trial included 94 patients (mean age, 62.1 years; 64.9% women) with previously treated locally advanced or metastatic squamous cell carcinoma of the anal canal. All patients progressed on or were intolerant of platinum-based chemotherapy, and nine (9.6%) had known HIV infection at baseline.

Patients received retifanlimab dosed at 500 mg via IV every 4 weeks. ORR assessed by independent central review served as the primary endpoint.

Results showed an ORR of 14%, with a 9.5-month (95% CI, 4.4-not estimable) median duration of response.

“Patients with [squamous cell carcinoma of the anal canal] who have progressed after first-line chemotherapy currently do not have approved treatment options,” Hervé Hoppenot, CEO of Incyte, said in the release. “[Although] we are not surprised with the FDA decision given the ODAC recommendation, we are disappointed. We remain committed to advancing science to find solutions for patients with unmet medical needs, and we will ensure close coordination with the FDA in order to address feedback and determine next steps for the review of retifanlimab.”

Common adverse events in the PODIUM-202 trial included fatigue (any grade, 38%; grade 3/grade 4, 6%) and diarrhea (any grade, 20%; grade 3/grade 4, 3.2%).

Retifanlimab exhibited a safety profile consistent with that of approved immune checkpoint inhibitors, although an FDA briefing document stated that because of the small size of the safety database and the trial’s single-arm design, “residual uncertainty remains regarding the risks in the indicated patient population.”