Abstract
BackgroundIn the primary analysis of the ERIVANCE BCC trial, vismodegib, the first US Food and Drug Administration–approved Hedgehog pathway inhibitor, showed objective response rates (ORRs) by independent review facility (IRF) of 30% and 43% in metastatic basal cell carcinoma (mBCC) and locally advanced BCC (laBCC), respectively. ORRs by investigator review were 45% (mBCC) and 60% (laBCC). Herein, we present long-term safety and final investigator-assessed efficacy results in patients with mBCC or laBCC.MethodsOne hundred four patients with measurable advanced BCC received oral vismodegib 150 mg once daily until disease progression or intolerable toxicity. The primary end point was IRF-assessed ORR. Secondary end points included ORR, duration of response (DOR), progression-free survival, overall survival (OS), and safety.ResultsAt data cutoff (39 months after completion of accrual), 8 patients were receiving the study drug (69 patients in survival follow-up). Investigator-assessed ORR was 48.5% in the mBCC group (all partial responses) and 60.3% in the laBCC group (20 patients had complete response and 18 patients had partial response). ORRs were comparable across patient subgroups, including aggressive histologic subtypes (eg, infiltrative BCC). Median DOR was 14.8 months (mBCC) and 26.2 months (laBCC). Median OS was 33.4 months in the mBCC cohort and not estimable in the laBCC cohort. Adverse events remained consistent with clinical experience. Thirty-three deaths (31.7%) were reported; none were related to vismodegib.ConclusionsThis long-term update of the ERIVANCE BCC trial demonstrated durability of response, efficacy across patient subgroups, and manageable long-term safety of vismodegib in patients with advanced BCC.Trial registrationThis study was registered prospectively with Clinicaltrials.gov, number NCT00833417 on January 30, 2009.
Highlights
In the primary analysis of the ERIVANCE Basal cell carcinoma (BCC) trial, vismodegib, the first US Food and Drug Administration–approved Hedgehog pathway inhibitor, showed objective response rates (ORRs) by independent review facility (IRF) of 30% and 43% in metastatic basal cell carcinoma and locally advanced BCC, respectively
Vismodegib, a first-in-class small molecule inhibitor of Hedgehog pathway signaling [11,12,13], was approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of adults with metastatic basal cell carcinoma (mBCC) or with locally advanced BCC (laBCC) that has recurred after surgery or who are not candidates for surgery or radiation [14, 15]
We report final data from ERIVANCE BCC, with 39 months of follow-up after the completion of accrual, that confirms and extends the long-term safety and durability of response associated with vismodegib and further evaluates efficacy across relevant patient subgroups and tumor histologic subtypes
Summary
In the primary analysis of the ERIVANCE BCC trial, vismodegib, the first US Food and Drug Administration–approved Hedgehog pathway inhibitor, showed objective response rates (ORRs) by independent review facility (IRF) of 30% and 43% in metastatic basal cell carcinoma (mBCC) and locally advanced BCC (laBCC), respectively. Vismodegib, a first-in-class small molecule inhibitor of Hedgehog pathway signaling [11,12,13], was approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of adults with mBCC or with laBCC that has recurred after surgery or who are not candidates for surgery or radiation [14, 15]. A second Hedgehog pathway inhibitor (HPI) (sonidegib) has been approved for laBCC based on the results from the BOLT study [16]
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