Advancements in the treatment of geographic atrophy: focus on pegcetacoplan in age-related macular degeneration

Ann Med Surg (Lond). 2023 Nov 1;85(12):6067-6077. doi: 10.1097/MS9.0000000000001466. eCollection 2023 Dec.

Abstract

Geographic atrophy (GA) is a progressive form of age-related macular degeneration characterized by the degeneration of retinal pigment epithelial cells and photoreceptor death. The dysregulation of the complement cascade has been implicated in GA progression. This review provides a comprehensive overview of the pathophysiology of age-related macular degeneration and GA, discusses current therapeutic options, and focuses on the recent breakthrough drug, pegcetacoplan (SYFOVRE). Pegcetacoplan is a complement inhibitor that selectively targets the C3 complement protein, effectively modulating complement activation. Clinical trials, including the OAKS and DERBY studies, have demonstrated the efficacy of SYFOVRE in reducing the growth of GA lesions compared to placebo. The FDA approval of SYFOVRE as the first and only definitive therapy for GA marks a significant milestone in the management of this debilitating condition. The review also explores potential future treatment strategies, including immune-modulating agents and ocular gene therapy. While SYFOVRE offers new hope for GA patients, further research is needed to evaluate its long-term benefits, safety profile, and optimal treatment regimens.

Keywords: SYFOVRE; age-related macular degeneration; complement cascade; geographic atrophy; pegcetacoplan; photoreceptor death; retinal pigment epithelium.

Publication types

  • Review