Pivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC

      Background

      Primary analysis from the pivotal ERIVANCE BCC study resulted in approval of vismodegib, a Hedgehog pathway inhibitor indicated for treatment of adults with metastatic or locally advanced basal cell carcinoma (BCC) that has recurred after surgery or for patients who are not candidates for surgery or radiation.

      Objective

      An efficacy and safety analysis was conducted 12 months after primary analysis.

      Methods

      This was a multinational, multicenter, nonrandomized, 2-cohort study in patients with measurable and histologically confirmed locally advanced or metastatic BCC taking oral vismodegib (150 mg/d). Primary outcome measure was objective response rate (complete and partial responses) assessed by independent review facility.

      Results

      After 12 months of additional follow-up, median duration of exposure to vismodegib was 12.9 months. Objective response rate increased from 30.3% to 33.3% in patients with metastatic disease, and from 42.9% to 47.6% in patients with the locally advanced form. Median duration of response in patients with locally advanced BCC increased from 7.6 to 9.5 months. No new safety signals emerged with extended treatment duration.

      Limitations

      Limitations include low prevalence of advanced BCC and challenges of designing a study with heterogenous manifestations.

      Conclusion

      The 12-month update of the study confirms the efficacy and safety of vismodegib in management of advanced BCC.

      Key words

      Abbreviations used:

      AE ( adverse event), BCC ( basal cell carcinoma), CI ( confidence interval), DOR ( duration of response), FDA ( US Food and Drug Administration), IRF ( independent review facility), laBCC ( locally advanced BCC), mBCC ( metastatic BCC), PFS ( progression-free survival), TEAE ( treatment-emergent adverse event)
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