Pegcetacoplan Demonstrates Long-Term Benefit in Geographic Atrophy treatment
Table of Contents
Recent data presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting and further reinforced at the American Society of Retinal Specialists (ASRS) 2025 meeting, demonstrates that pegcetacoplan (Syfovre®) provides sustained benefit in slowing the progression of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The data, stemming from the pivotal DERBY and OAKS trials and their ongoing GALE open-label extension study, highlights the importance of early and continuous treatment to maximize retinal tissue preservation and visual function. https://www.syfovre.com/
Primary Topic: Geographic Atrophy (GA) and Pegcetacoplan Treatment
Primary Keyword: Pegcetacoplan
Understanding Geographic Atrophy
Geographic atrophy is an advanced form of age-related macular degeneration (AMD) that affects the macula, the central part of the retina responsible for sharp, central vision. In GA, cells in the macula progressively die, leading to irreversible vision loss. Unlike the “wet” form of AMD,which can sometimes be treated with anti-VEGF injections,GA has historically lacked effective treatment options. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration/geographic-atrophy
Pegcetacoplan (Syfovre®): A Novel Treatment Approach
Pegcetacoplan is a first-in-class complement C5 inhibitor designed to target and block the overactive complement cascade, a key driver of inflammation and retinal cell death in GA. The complement system is part of the immune system, but in AMD, it becomes dysregulated and attacks healthy retinal tissue. By inhibiting C5,pegcetacoplan aims to reduce this destructive process. https://www.aao.org/eye-health/diseases/amd-geographic-atrophy-syfovre
Key Findings from DERBY, OAKS, and GALE Studies
The DERBY and OAKS Phase 3 clinical trials demonstrated the efficacy of pegcetacoplan in reducing GA area progression over 12 months.The ongoing GALE open-label extension study provides longer-term data, showing sustained benefits with continued treatment.
Long-Term Tissue Preservation
Data presented at ASRS 2025, building on previous ARVO 2024 presentations by Roger A. Goldberg, MD, MBA, from Bay Area Retina Associates, reveals significant retinal tissue preservation over 4 years. Specifically:
Monthly Treatment: Preserved an average of 3.16 square millimeters of retinal tissue over 4 years.
Every-Other-Month Treatment: Preserved an average of 2.77 square millimeters of retinal tissue over 4 years.
* Treatment Delay: Patients who delayed treatment for 2 years only preserved 1.1 square millimeters of retinal tissue, underscoring the importance of early intervention.
Microperimetry Results & Visual Function
Microperimetry,a technique used to measure retinal sensitivity,showed a 35% risk reduction in early-treated patients for progression of absolute scotoma (blind spots) in critical areas of central vision (4 and 16 loci). This suggests that pegcetacoplan not only preserves retinal tissue but also helps maintain visual function.
Safety Profile and Real-world Experience
Pegcetacoplan has demonstrated a favorable safety profile in clinical trials and real-world use. Over 700,000 injections have been administered globally, with more than 100,000 new treatment starts, providing a robust safety dataset. Common adverse events are generally mild and manageable, including eye pain, conjunctival hemorrhage, and increased intraocular pressure.https://www.healio.com/ophthalmology/retina-vitreous/news/online-exclusive/syfovre-demonstrates-long-term-benefit-in-geographic-atrophy
The Importance of Early Intervention
the data consistently emphasizes