Vemurafenib in melanoma.

The incidence of malignant melanoma is increasing annually. Early stages can be cured with surgical intervention but metastatic disease has generally had a dismal prognosis with few effective interventions. A half of all melanomas possess a BRAF mutation, which can be targeted by specific inhibitors. Vemurafenib is an orally active, purposely designed mutant BRAF inhibitor, which has recently been shown to have a survival benefit measured in months in metastatic patients. In this article, the authors discuss the scientific rationale, drug development process and clinical trials that have led to vemurafenib becoming the first BRAF inhibitor approved for the treatment of patients with mutant BRAF metastatic melanoma.