A Comprehensive Monograph on Verteporfin (Visudyne®)

Section 1: Introduction and Drug Profile

1.1 Overview and Therapeutic Context

Verteporfin is a synthetic, second-generation benzoporphyrin derivative monoacid (BPD-MA), a small molecule photosensitizing agent that has carved out a significant and evolving role in modern therapeutics.[1] Marketed under the trade name Visudyne®, it is clinically supplied as a liposomal formulation designed for intravenous administration.[1] Its primary and most established application is in the field of ophthalmology as the active agent in photodynamic therapy (PDT), a modality that utilizes light to achieve targeted tissue destruction.[1] Specifically, Verteporfin PDT was developed for the selective elimination of abnormal blood vessels in the choroid, the vascular layer of the eye, which is the hallmark of conditions such as the exudative (wet) form of age-related macular degeneration (wet AMD).[1]

Beyond its foundational role as a light-activated drug, a deeper understanding of its molecular interactions has revealed a second, distinct pharmacological identity. Verteporfin has been identified as a potent, light-independent inhibitor of critical intracellular signaling pathways, most notably the Hippo-YAP pathway.[7] This discovery has catalyzed a wave of investigational research, repositioning Verteporfin as a candidate for entirely new therapeutic applications, including oncology and the burgeoning field of regenerative medicine.[4] Its journey from a specialized ophthalmic treatment to a potential multi-purpose pathway modulator illustrates a compelling narrative of scientific discovery and drug repurposing.

1.2 Identification and Chemical Properties