Pegaptanib Sodium in the Treatment of Ocular Vascular Diseases: Expectations and Realities

Age related macular degeneration (AMD) and proliferative diabetic retinopathy (PDR) are two of the most dreadful ocular vascular diseases that often result in severe visual impairment, placing significant burdens on the health care. In the recent years, both AMD and PDR have garnered a great deal of attention in the ophthalmic community because newly available treatment modalities have drastically improved the previously poor prognosis associated with these diseases.

Toward the end of the last century, the focus of the treatment for exudative AMD had shifted from laser photocoagulation to photodynamic therapy with Verteporfin, and then to the current uses of antiangiogenic agents, which block the vascular endothelial growth factor (VEGF). Similarly, the Early Treatment Diabetic Retinopathy Study and the Diabetic Retinopathy Study had established laser photocoagulation as a widely accepted therapeutic strategy in PDR. However, recent data have suggested possible benefits of VEGF blocking agents in the treatment of PDR.

Pegaptanib sodium (Macugen®; Eyetech Inc., New York, NY, USA) is a selective RNA aptamer that inhibits the 165 isomer of the VEGF-A molecule. This drug was the first VEGF inhibitor molecule approved by the United States Food and Drug Administration for the treatment of ocular vascular diseases. Several studies over the past years have demonstrated the safety and efficacy of pegaptanib sodium in the treatment of both AMD and PDR. In this review we will examine the promises and expectations bestowed on pegaptanib by the medical community on its launch and the degree to which these have been met in the treatment of AMD and PDR.

Keywords: Pegaptanib Sodium, ocular vascular diseases, age related macular degeneration (AMD), proliferative diabetic retinopathy (PDR)