Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease

Nat Rev Drug Discov. 2006 Feb;5(2):123-32. doi: 10.1038/nrd1955.


Aptamers are oligonucleotide ligands that are selected for high-affinity binding to molecular targets. Pegaptanib sodium (Macugen; Eyetech Pharmaceuticals/Pfizer) is an RNA aptamer directed against vascular endothelial growth factor (VEGF)-165, the VEGF isoform primarily responsible for pathological ocular neovascularization and vascular permeability. After nearly a decade of preclinical development to optimize and characterize its biological effects, pegaptanib was shown in clinical trials to be effective in treating choroidal neovascularization associated with age-related macular degeneration. Pegaptanib therefore has the notable distinction of being the first aptamer therapeutic approved for use in humans, paving the way for future aptamer applications.

Publication types

  • Review

MeSH terms

  • Angiogenesis Inhibitors / pharmacokinetics
  • Angiogenesis Inhibitors / pharmacology
  • Angiogenesis Inhibitors / therapeutic use*
  • Animals
  • Aptamers, Nucleotide / pharmacokinetics
  • Aptamers, Nucleotide / pharmacology
  • Aptamers, Nucleotide / therapeutic use*
  • Clinical Trials as Topic
  • Diabetic Retinopathy / drug therapy
  • Drug Delivery Systems
  • Eye / blood supply
  • Eye Diseases / drug therapy*
  • Humans
  • Neovascularization, Pathologic / drug therapy*
  • Vascular Endothelial Growth Factor A / antagonists & inhibitors*


  • Angiogenesis Inhibitors
  • Aptamers, Nucleotide
  • Vascular Endothelial Growth Factor A
  • pegaptanib