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The therapy is the first and only FDA-approved treatment for geographic atrophy, with approval based on positive results from the phase 3 DERBY and OAKS trials.
The US Food and Drug Administration (FDA) has approved pegcetacoplan injection (SYFOVRE™) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
It marks the first and only FDA-approved treatment for GA, a leading cause of blindness than impacts more than one million people in the United States. The approval for pegcetacoplan was announced by Apellis Pharmaceuticals, Inc in a news release.
“The approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, MD, PhD, director of ophthalmology clinical research, Duke University Medical Center. “Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined. With SYFOVRE, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”
Pegcetacoplan injection is now approved for patients with GA with or without subfoveal involvement and provides dosing flexibility for patients and physicians with a dosing regimen of every 25 to 60 days. By targeting C3, pegcetacoplan is designed to provide comprehensive control of the complement cascade.
The therapy's approval was based on positive results from the Phase 3 OAKS and DERBY trials at 24 months across a representative population of patients. In the OAKS and DERBY studies, pegcetacoplan injection reduced the rate of GA lesion growth compared to sham and demonstrated increased treatment effects over time. Data showed the greatest benefit of up to 36% reduction in lesion growth with monthly treatment in DERBY and occurred between months 18 to 24.
Trial results additionally suggested a well-demonstrated safety profile following approximately 12,000 injections. Results indicate the most common adverse reactions reported in patients receiving pegcetacoplan injection were ocular discomfort, neovascular AMD, vitreous floaters, and conjunctival hemorrhage.
An advanced form of AMD, GA is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. The disease can severely impair quality of life by making it difficult to engage in daily activities and could take only 2.5 years for GA lesions to start impacting the fovea.
“For the first time ever, we are celebrating the approval of a treatment for GA,” said Jeff Todd, president, and chief executive officer, of Prevent Blindness in the release. “This is a historic and hopeful day for all GA patients and their care partners, who have been waiting for treatment for this relentless form of vision loss.”
At the American Academy of Ophthalmology (AAO) 2022 Annual Meeting in Chicago, Illinois, Rishi Singh, MD, Cleveland Clinic Florida, discussed the significance of pegcetacoplan for GA treatment and the high burden faced by patients with the disease.
"This drug potentially represents a way of reducing that burden of those patients," Singh told HCPLive.2 "And it's really going to be, I think, transformative for those patients who can receive this drug and receive it in a timely fashion."
Singh additionally noted how the therapy may ultimately provide a better understanding of the importance of the complement pathway in preventing the progression of the disease.
“This is the first of many therapies to come down that area, and this is going to lead to hopefully more sustained therapies, more durable therapies, and potentially more effective therapies over time,” Singh said.
1. FDA approves SYFOVRE™ (Pegcetacoplan Injection) as the first and only treatment for geographic atrophy (GA), a leading cause of blindness. Apellis Pharmaceuticals, Inc. https://investors.apellis.com/news-releases/news-release-details/fda-approves-syfovretm-pegcetacoplan-injection-first-and-only. Published February 17, 2023.
2. Iapoce C. Rishi Singh, MD: Efficacy of pegcetacoplan for GA based on 24-month data. HCP Live. https://www.hcplive.com/view/rishi-singh-md-efficacy-pegcetacoplan-for-ga-24-month-data. Published October 2, 2022.