Pegaptanib: the first antiangiogenic agent approved for neovascular macular degeneration.

Age-related macular degeneration is the leading cause of irreversible visual loss in the industrialised world. Angiogenesis underlies the neovascularisation, and vascular endothelial growth factor (VEGF) is an angiogenesis growth factor. In the VEGF Inhibition Study in Ocular Neovascularisation-1 (VISION-1) trial, pegaptanib (an aptamer inhibitor of VEGF) was tested in neovascular age-related macular degeneration. The 1186 patients received a sham injection or intravitreous injection of pegaptanib (0.3, 1.0 or 3.0 mg) every 6 weeks over a period of 48 weeks. The primary end point was the proportion of patients who lost < 15 letters of visual acuity between baseline and 54 weeks, and this occurred in 164/296 patients (55%) who received the sham injection. A higher percentage of patients maintained this visual acuity if they were treated with pegaptanib 0.3 mg (54/206 patients, 70%). There was no evidence that pegaptanib 1 or 3 mg was more effective than 0.3 mg. There was no excess of systemic adverse effects with pegaptanib, but ocular adverse effects occurred more commonly with pegaptanib than with sham injection; vitreous floaters (33 versus 8%), vitreous opacities (18 versus 10%) and anterior chamber inflammation (14 versus 6%). Although these results represent a new, beneficial and relatively safe approach to age-related macular degeneration, the progression was not halted or reversed, and further improvement to treatment for this condition should be sought.