FDA Approves Pegcetacoplan Injection for Geographic Atrophy

Officials with the FDA have approved pegcetacoplan injection (Syfovre; Apellis Pharmaceuticals) as the first and only treatment for geographic atrophy (GA) secondary to age-related macular degeneration.

GA is a leading cause of blindness, impacting more than 1 million individuals in the United States and 5 million individuals worldwide. It is an advanced form of age-related macular degeneration and is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision.

By targeting C3, pegcetacoplan is designed to provide comprehensive control of the complement cascade.

“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, MD, PhD, lead investigator for the OAKS study, in a press release. “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.”

The approval was based on results from the phase 3 OAKS and DERBY studies at 24 months across a broad and representative population of patients. In these studies, pegcetacoplan reduced the rate of GA lesion growth compared to sham and demonstrated increasing treatment effects over time, with the greatest benefit (up to 36% reduction in lesion growth with monthly treatment in DERBY) occurring between months 18 to 24.

Both monthly and every-other-month treatment with pegcetacoplan reduced the rate of GA lesion growth through 24 months compared to sham. In the OAKS trial, monthly injections reduced growth by 22% whereas every-other-month injections reduced growth by 18%. Similarly, the DERBY trial saw 18% reduced growth with monthly dosing and 17% reduced growth with every-other-month dosing.

“Today marks an extraordinary milestone for patients, the retina community, and Apellis,” said Cedric Francois, MD, PhD, co-founder and CEO of Apellis, in the press release. “With its increasing effects over time and flexible dosing, we believe that Syfovre will make a meaningful difference in the lives of people with GA. GA is a complex disease that the field has spent decades trying to address, so we are humbled and proud to bring forward the first-ever treatment.”

Pegcetacoplan is approved for patients with GA, with or without subfoveal involvement, and provides dosing flexibility with a regimen of every 25 to 60 days. The safety profile is well-demonstrated following approximately 12,000 injections. The most common adverse events reported in patients receiving pegcetacoplan were ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, and conjunctival hemorrhage.

“For the first time ever, we are celebrating the approval of a treatment for GA,” said Jeff Todd, president and CEO at Prevent Blindness, in the press release. “This is a historic and hopeful day for all GA patients and their care partners, who have been waiting for a treatment for this relentless form of vision loss.”

REFERENCE

FDA Approves Syfovre (pegcetacoplan injection) as the First and Only Treatment for Geographic Atrophy (GA), a Leading Cause of Blindness. News release. Apellis; February 17, 2023. Accessed February 20, 2023. https://investors.apellis.com/news-releases/news-release-details/fda-approves-syfovretm-pegcetacoplan-injection-first-and-only