Pegaptanib[2] sodium injection (brand name Macugen) is an anti-angiogenic medicine for the treatment of neovascular (wet) age-related macular degeneration (AMD).

[5] Pegaptanib is a pegylated anti-vascular endothelial growth factor (VEGF) aptamer, a single strand of nucleic acid that binds with specificity to a particular target.

Pegaptanib specifically binds to the 165 isoform of VEGF, a protein that plays a critical role in angiogenesis (the formation of new blood vessels) and increased permeability (leakage from blood vessels), two of the primary pathological processes responsible for the vision loss associated with neovascular AMD.

This then reduces the growth of the blood vessels located within the eye and works to control the leakage and swelling.

[3] Pegaptanib underwent several preclinical studies in order to determine its safety and efficacy before moving into clinical trials.

It was found that using poly(lactic-co-glycolic) acid (PLGA) microspheres, which encapsulated the drug, the minimum dosing frequency was 6 weeks to maintain the desired pharmacological effect.

In this study, 21 patients with subfoveal choroidal neovascularization (CNV) secondary to AMD were given multiple intravitreal injections.

Due to the presence of subfoveal CNV some patients were given a secondary treatment, photodynamic therapy (PDT) for this condition.

In groups treated with the 0.3 mg dose at the primary efficacy endpoint, experienced a statistically significant result.