The Food and Drug Administration (FDA)’s Oncologic Drugs Advisory Committee (ODAC) voted 13 to 4 to defer a regulatory decision on retifanlimab, an investigational PD-1 inhibitor for locally advanced or metastatic squamous cell carcinoma of the anal canal (SCAC), until further data from a confirmatory trial are made available.

The Biologics License Application (BLA) submission for retifanlimab included data from an open-label, single-arm phase 2 trial (POD1UM-202; Identifier: NCT03597295), which evaluated retifanlimab in 94 adults with locally advanced or metastatic SCAC who progressed after platinum-based chemotherapy.

Findings from this trial showed an overall response rate (ORR) of 13.8% (95% CI, 7.6-22.5), with 1 patient achieving complete response, 12 with partial responses and 33 with stable disease. Disease control rate, defined as maintaining either ORR or stable disease, was confirmed to be 49%. Responses were observed regardless of PD-L1 status, presence of liver metastases, age or HIV+ status and were durable (median 9.5 months). 

In the ongoing, phase 3 confirmatory trial (POD1UM-303; Identifier: NCT04472429), retifanlimab is being evaluated in adults with inoperable locally recurrent or metastatic SCAC who have not previously received platinum-based chemotherapy.

“While we are disappointed by the outcome of today’s ODAC vote, we will continue to work closely with the FDA as it completes its review of the BLA for retifanlimab,” said Lance Leopold, MD, Group Vice President, Immuno-Oncology Clinical Development, Incyte. “We continue to believe that retifanlimab can provide an additional, much-needed option for these patients based on the favorable benefit/risk shown in our trial.”

Although not bound by the committee’s recommendations, the Agency does take them into consideration when making decisions on approval. A Prescription Drug User Fee Act (PDUFA) target date of July 25, 2021 has been set for the application.


Incyte announces outcome of FDA Oncologic Drugs Advisory Committee (ODAC) meeting reviewing retifanlimab as a treatment for patients with squamous cell carcinoma of the anal canal (SCAC). [press release]. Wilmington, DE: Incyte; June 24, 2021.