Landmark clinical trial data for FDA-approved immunotherapies for CSCC
| Trial characteristics | Outcomes for FDA approval | ||||
| Trial | Study design | Study population for SBLA | Intervention(s) | ORR | Median DOR |
| Study 1423 (NCT02383212) and Study 1540/EMPOWER CSCC 1* (NCT02760498)7 | Study 1423: phase I open-label, non-randomized Study 1540: phase II open-label, non-randomized | Metastatic or locally advanced CSCC not eligible for curative surgery or radiation (Study 1423, n=26; Study 1540, n=82) | Cemiplimab | 47.2% (95% CI 37.5% to 57.1%)†‡ | Not reached (range 1.0–15.2+ months)†‡ |
| KEYNOTE-629§ (NCT03284424)8 | Phase II open-label, non-randomized | Metastatic or recurrent CSCC not eligible for curative surgery or radiation (n=105) | Pembrolizumab | 34% (95% CI 25% to 44%)† | Not reached (range 2.7–13.1+ months)† |
| Locally advanced CSCC not eligible for curative surgery or radiation (n=54) | 50% (95% CI 36% to 64%)† | Not reached (range 1.0+ to 17.2+ months)† | |||
*In EMPOWER CSCC 1/Study 1540, patients with CSCC not eligible for surgery or radiation with no nodal or distant metastases were defined as ‘locally advanced’. Patients with any nodal or distant metastases were defined as ‘metastatic’.152 153
†Since approval, updated follow-up results have become available at the time of guideline publication, which are discussed in the narrative text.
‡Data presented are from the combined analysis of patients with metastatic and locally advanced CSCC who received cemiplimab at 3 mg/kg IV every 2 weeks.
§In KEYNOTE-629, patients with CSCC ineligible for surgery or radiation or had prior radiation were defined as locally advanced. Patients with locoregionally recurrent disease (including locoregional lymph node metastases) not curable by surgery or radiation and/or distant metastatic disease (disseminated disease distant to the initial primary site of diagnosis) were defined as ‘recurrent/metastatic.’111 112
CI, confidence interval; CSCC, cutaneous squamous cell carcinoma; DOR, duration of response; FDA, Food and Drug Administration; ORR, objective response rate; SBLA, Supplemental Biologics License Application.