Basal cell carcinoma (BCC) is the most common periocular skin cancer and can lead to significant morbidity. We assess the effectiveness of vismodegib, a first-in-class Hedgehog signaling pathway inhibitor, in the management of periocular and orbital BCCs based on clinical response, tolerability, and orbital content preservation. All patients with periocular or orbital BCCs who met criteria for ...

Purpose: To investigate the safety, dose-limiting toxicities, and pharmacokinetics of the smoothened inhibitor vismodegib in children with refractory or relapsed medulloblastoma. Experimental design: Initially, vismodegib was administered daily at 85 mg/m and escalated to 170 mg/m. The study was then revised to investigate a flat-dosing schedule of 150 mg for patients with small body surface ar...

Because many patients with unresectable basal cell carcinoma (BCC) are aged ≥65 years, this study explores the efficacy and safety of vismodegib in these patients with locally advanced (la) or metastatic (m) basal cell carcinoma (BCC) in the ERIVANCE BCC trial and the expanded access study (EAS).We compared patients aged ≥65 years to patients aged <65 years taking vismodegib 150 mg/day, using d...

In January 2012, vismodegib (Erivedge, manufactured by Genentech) became the first selective inhibitor of the Hedgehog signaling pathway to be approved by the US Food and Drug Administration for the treatment of locally advanced and metastatic basal cell carcinoma. The drug selectively binds to Smoothened, a 7-helix transmembrane receptor, thereby inhibiting activation of transcription factors ...

SHORT COMMUNICATION Vismodegib, a Hedgehog (Hh) pathway inhibitor, was approved by the US Food and Drug Administration (FDA) in 2012 for the treatment of locally advanced (la) and metastatic (m) basal cell carcinoma (BCC). Interim results of the largest clinical study of vismodegib have shown complete (17.5%) or partial (39.8%) response, and stable (39.0%) or progressive (2.8%) disease in 251 c...

Vismodegib (Erivedge) for advanced basal cell carcinoma.

BCC: Basal cell carcinoma INTRODUCTION The case of a 65-year-oldwomanwith an advanced, ulcerated biopsy-proven basal cell carcinoma (BCC) located on the left side of her upper back is reviewed. She was treated with vismodegib with only mild clinical improvement. Pathologic analysis of subsequent surgical resection found only granulation tissue with no evidence of residual tumor. It is important...

Vismodegib (GDC-0449, 2-chloro-N-(4-chloro-3-(pyridin-2-yl)phenyl)-4-(methylsulfonyl)benzamide, Erivedge™) is a novel first-in-human, first-in class, orally bio-available Hedgehog pathway signaling inhibitor of the G-protein coupled receptor-like protein smoothened (SMO) which was approved in the United States on January 2012. This signaling pathway is involved in the carcinogenesis of several ...

AGEP: acute generalized exanthematous pustulosis BCC: basal cell carcinoma SJS/TEN: Stevens-Johnson syndrome/toxic epidermal necrolysis INTRODUCTION Basal cell carcinoma (BCC) is a common skin neoplasm that usually has a nonaggressive behavior typically managed with local treatment (eg, surgery, photodynamic treatment, topical agents, or radiation). In some cases, BCC has a much more invasive b...

Medulloblastoma is a cancer of the cerebellum, for which there is currently no approved targeted therapy. Recent transcriptomics approaches have demonstrated that medulloblastoma is composed of molecularly distinct subgroups, one of which is characterized by activation of the Hedgehog pathway, which in mouse models is sufficient to drive medulloblastoma development. There is thus considerable i...