Table 2

Landmark clinical trial data for FDA-approved immunotherapies for MCC

Trial characteristicsOutcomes for FDA approval
TrialStudy designStudy population for SBLAIntervention(s)ORRMedian DOR
JAVELIN Merkel 200 (NCT02155647)6 57Phase II open-label, non-randomizedPart A: metastatic, chemotherapy R/R MCC (n=88)Avelumab33.0% (95% CI 23.3% to 43.8%)*Median not reached (range 2.8–23.3+ months)*
Part B†: metastatic, systemic therapy naïve MCC (n=116)39.7% (95% CI 30.7% to 49.2%)18.2 months (95% CI 11.3 to NE)
KEYNOTE-017 (NCT02267603)8Phase II open-label, non-randomizedRecurrent locally advanced or metastatic MCC (n=50)Pembrolizumab56% (95% CI 41% to 70.0%)*Median not reached (range 5.9–34.5+ months)*
  • *Since approval, updated follow-up results have become available, which are discussed in the Approved anti-PD-(L)1 agents for MCC section.

  • †Data from this cohort were not evaluated for the FDA approval of avelumab.

  • CI, confidence interval; DOR, duration of response; FDA, Food and Drug Administration; MCC, Merkel cell carcinoma; NE, not estimable; ORR, objective response rate; PD-(L)1, PD-1/PD-L1 axis; R/R, relapsed/refractory; SBLA, Supplemental Biologics License Application.