Pegaptanib: vascular endothelial growth factor inhibition in age-related macular degeneration

Age-related macular degeneration causes blindness in elderly individuals. In the last decade, the search for mechanisms that initiate and sustain neovascularization in age-related macular degeneration has focused on the actions of vascular endothelial growth factor. The first approved drug that inhibited vascular endothelial growth factor for the treatment of neovascular age-related macular degeneration was pegaptanib sodium. This pegylated RNA aptamer has shown efficacy in two pivotal Phase III trials for the treatment of neovascular age-related macular degeneration. Pegaptanib therefore has the notable distinction of being the only aptamer therapeutic approved for use in humans, possibly paving the road for future aptamer applications.