Vismodegib ( Erivedge ), a first-in-class Hedgehog pathway inhibitor, was Food and Drug Administration ( FDA ) approved for advanced basal cell carcinomas ( BCCs ) based on a single, nonrandomized, phase-II trial. Consequently, additional clinical data are critical to confirm the efficacy and safety of Vismodegib.
Researchers have assessed the efficacy and the safety of Vismodegib, while providing early drug access to patients with advanced basal cell carcinoma and limited treatment options.
This was an open-label, multicenter study in patients with advanced basal cell carcinoma inappropriate for radiotherapy or surgery.
Patients received 150 mg Vismodegib daily until disease progression or intolerable toxicity. Tumor response was assessed via Response Evaluation Criteria in Solid Tumors version 1.0.
A total of 119 patients with advanced basal cell carcinoma took Vismodegib for a median of 5.5 months. Objective responses occurred in 46.4% of locally advanced basal cell carcinoma and 30.8% of patients with metastatic basal cell carcinoma.
Response was negatively associated with prior systemic therapy in patients with locally advanced basal cell carcinoma ( P=0.002 ).
Mean follow-up for safety was 6.5 months, with muscle spasms ( 70.6% ), dysgeusia ( 70.6% ), alopecia ( 58.0% ), and diarrhea ( 25.2% ) as the most common adverse events.
The study has provided important clinical data supporting the efficacy and safety of Vismodegib. Larger studies are underway to assess predictors of response and long-term outcomes. ( Xagena )
Chang ALet al, J Am Acad Dermatol 2014; 70: 60-69