Retifanlimab (Incyte), an investigational intravenous PD-1 inhibitor, has been granted Priority Review by the Food and Drug Administration (FDA) for the treatment of adults with locally advanced or metastatic squamous cell carcinoma of the anal canal (SCAC) who have progressed on, or who are intolerant of, platinum-based chemotherapy.

The BLA submission is supported by data from the open-label, single-arm phase 2 POD1UM-202 trial that evaluated the efficacy of retifanlimab in 94 adults with locally advanced or metastatic SCAC who progressed after platinum-based chemotherapy. Patients received retifanlimab 500mg intravenously every 4 weeks; the primary end point was objective response rate (ORR). 

Findings showed an ORR of 13.8% (95% CI, 7.6-22.5), as determined by independent central review using RECIST v1.1; responses include 1 complete response, 12 partial responses and 33 stable disease. Additionally, 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). 

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As for safety, the most common adverse reactions (incidence of greater than or equal to 20%) were fatigue and diarrhea. Treatment-related and immune-related adverse events of grade 3 or greater occurred in 11.7% and 6.4% of patients, respectively.

“Patients with SCAC who have progressed after first-line chemotherapy treatment currently have no approved treatments available, and we are encouraged that the FDA’s acceptance of this BLA for Priority Review brings us one step closer to addressing this historically neglected, yet important, tumor,” said Lance Leopold, MD, Group Vice President, Immuno-Oncology Clinical Development, Incyte.

A Prescription Drug User Fee Act (PDUFA) target date of July 25, 2021 has been set for this application. The Company is also evaluating retifanlimab, in combination with carboplatin and paclitaxel, in a phase 3 trial for the treatment of inoperable locally recurrent or metastatic SCAC.


1. Incyte announces acceptance and Priority Review of BLA for retifanlimab as a potential treatment for patients with squamous cell carcinoma of the anal canal (SCAC). [press release]. Wilmington, DE: Incyte; January 21, 2021. 

2. Incyte announces encouraging results from phase 2 trial of retifanlimab (INCMGA0012) in patients with previously treated, advanced squamous cell carcinoma of the anal canal. [press release]. Wilmington, DE: Incyte; September 18, 2020.

This article originally appeared on MPR