FDA Approves First Treatment for Geographic Atrophy

The first ever treatment for geographic atrophy (GA) was approved by the FDA. Pegcetacoplan (Syfovre; Apellis Pharmaceuticals) was approved to treat GA secondary to age-related macular degeneration (AMD).

GA is a leading cause of blindness and it affects more than 1 million people in the United States and more than 5 million people worldwide. GA is an advanced form of dry AMD. While there are treatments for the wet form of AMD, there have been no available treatments for dry AMD until now.

“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” Eleonora Lad, MD, PhD, lead investigator for the OAKS study, director of ophthalmology clinical research, associate professor of ophthalmology, Duke University Medical Center, said in a statement.¹ “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 is based on the 24-month results of the DERBY (n = 621) and OAKS (n = 637) trials. These phase 3, multicenter, randomized, double-masked, sham-controlled trials studied and compared the efficacy of pegcetacoplan with sham injections. The studies evaluated both monthly injections and every other month injections.

According to Lad, there were reductions in lesions in both patients who received the drug monthly and every other month, although the “effect was stronger with monthly treatments.”²

The benefit of the 2 injection options is that they offer more flexibility to the patient regarding clinic visits. “In regards to safety, there were fewer new-onset exudations in the every other month group. So, this will be an important part of the conversation we as clinicians will have to have with our patients that we're considering treatment for,” she said.

In OAKS, the monthly injection reduced lesion growth rate by 22% compared with 18% for every other month. In DERBY, the monthly injection reduced lesion growth rate by 18% compared with 17% for every other month.

Lad added that pegcetacoplan’s ability to slow disease progression increased over time. “The fact that drug works better over time means it has a positive effect, and the patients should have the hope that they're preventing retinal degeneration more as they take the drug over longer time intervals,” she said.

The safety of pegcetacoplan is comparable to the data for other intravitreal injections.³ Over 24 months, the rate of infectious endophthalmitis was 0.034% per injection and the rate of intraocular inflammation was 0.24% per injection. There were no events of occlusive vasculitis or retinitis observed over 24 months, and no serious adverse events of ischemic optic neuropathy between months 18 and 24. The combined rate of new-onset exudations at month 24 was 11.9% for the monthly injection, 6.7% for the every other month injection, and 3.1% for the sham injection.

“Today marks an extraordinary milestone for patients, the retina community, and Apellis. 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,” said Cedric Francois, MD, PhD, cofounder and CEO, Apellis. “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.”

Reference

1. FDA Approves Syfovre™ (pegcetacoplan injection) as the first and only treatment for geographic atrophy (GA), a leading cause of blindness. News release. February 17, 2023. https://www.globenewswire.com/news-release/2023/02/17/2610967/0/en/FDA-Approves-SYFOVRE-pegcetacoplan-injection-as-the-First-and-Only-Treatment-for-Geographic-Atrophy-GA-a-Leading-Cause-of-Blindness.html

2. Joszt L. Effects of pegcetacoplan to slow progression of GA increase over time: Dr Eleonora Lad. The American Journal of Managed Care. February 10, 2023. Accessed February 17, 2023. https://www.ajmc.com/view/effects-of-pegcetacoplan-to-slow-progression-of-ga-increase-over-time-dr-eleonora-lad

3. Apellis announces 24-month results showing increased effects over time with pegcetacoplan in phase 3 DERBY and OAKS studies in geographic atrophy (GA). News release. August 24, 2022. Accessed February 17, 2023. https://investors.apellis.com/news-releases/news-release-details/apellis-announces-24-month-results-showing-increased-effects