Vemurafenib: the first drug approved for BRAF-mutant cancer

Nat Rev Drug Discov. 2012 Nov;11(11):873-86. doi: 10.1038/nrd3847. Epub 2012 Oct 12.

Abstract

The identification of driver oncogenes has provided important targets for drugs that can change the landscape of cancer therapies. One such example is the BRAF oncogene, which is found in about half of all melanomas as well as several other cancers. As a druggable kinase, oncogenic BRAF has become a crucial target of small-molecule drug discovery efforts. Following a rapid clinical development path, vemurafenib (Zelboraf; Plexxikon/Roche) was approved for the treatment of BRAF-mutated metastatic melanoma in the United States in August 2011 and the European Union in February 2012. This Review describes the underlying biology of BRAF, the technology used to identify vemurafenib and its clinical development milestones, along with future prospects based on lessons learned during its development.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Drug Approval
  • Drug Design
  • European Union
  • Humans
  • Indoles / pharmacology
  • Indoles / therapeutic use*
  • Melanoma / drug therapy*
  • Melanoma / genetics
  • Melanoma / pathology
  • Mutation
  • Neoplasm Metastasis
  • Proto-Oncogene Proteins B-raf / genetics
  • Skin Neoplasms / drug therapy*
  • Skin Neoplasms / genetics
  • Skin Neoplasms / pathology
  • Sulfonamides / pharmacology
  • Sulfonamides / therapeutic use*
  • Vereinigte Staaten
  • Vemurafenib

Substances

  • Antineoplastic Agents
  • Indoles
  • Sulfonamides
  • Vemurafenib
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf