Pegaptanib sodium for the treatment of ocular vascular disease

Pegaptanib sodium is a selective RNA aptamer that inhibits the 165 isoform of vascular endothelial growth factor (VEGF), a key regulator of pathological vascular growth and permeability and a contributor to vision loss in diseases such as age-related macular degeneration (AMD), diabetic retinopathy and retinal vein occlusion. The development of pegaptanib represented an important achievement in that it was both the first approved anti-VEGF agent for the treatment of ocular disease and the first clinically validated aptamer therapeutic. The safety and efficacy of pegaptanib for the treatment of neovascular AMD were established in the VEGF Inhibition Study in Ocular Neovascularization (VISION) trials. Investigations into the use of pegaptanib have now been expanded to include diabetic macular edema, macular edema secondary to central retinal vein occlusion and other ocular vascular diseases. Pegaptanib is also being used investigationally in combination with other agents.