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

Abstract

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.

• Authors: Eugene W M Ng, David T Shima, Perry Calias, Emmett T Cunningham Jr, David R Guyer, Anthony P Adamis
• Journal: Nature reviews. Drug discovery (Nat Rev Drug Discov) Vol. 5 Issue 2 Pg. 123-32 (Feb 2006) ISSN: 1474-1776 [Print] England
• PMID: 16518379 (Publication Type: Journal Article, Review)

Chemical References

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

Topics

• Angiogenesis Inhibitors (pharmacokinetics, pharmacology, therapeutic use)
• Animals
• Aptamers, Nucleotide (pharmacokinetics, pharmacology, 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)