The FDA has approved Apellis Pharmaceuticals' Syfovre (pegcetacoplan) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This marks the first time that patients with GA have an FDA-approved treatment option, which is a significant milestone in medical history.
“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 company news 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.”
Syfovre is expected to be available in the US by the beginning of March through specialty distributors and specialty pharmacies nationwide.
“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,” Cedric Francois, MD, PhD, co-founder and chief executive officer, Apellis, said in the news release. “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. Thank you to all who helped make this moment a reality.”
Syfovre's approval is supported by positive outcomes from the phase 3 OAKS and DERBY studies conducted over 24 months, which included a diverse group of patients. The treatment is FDA-approved for patients with geographic atrophy (GA), regardless of subfoveal involvement, and offers dosing flexibility with a regimen that can be administered every 25 to 60 days.
In the OAKS and DERBY studies, Syfovre was shown to decrease the rate of GA lesion growth in comparison to a placebo, and demonstrated an increasing treatment effect over time. The most significant benefit (up to a 36% reduction in lesion growth with monthly treatment in DERBY) was observed between months 18 and 24.
Syfovre has a well-demonstrated safety profile, with approximately 12,000 injections having been administered. Among patients receiving Syfovre, the most common adverse reactions (≥5%) reported were ocular discomfort, neovascular age-related macular degeneration (AMD), vitreous floaters, and conjunctival hemorrhage. Syfovre is not recommended for use in patients with ocular or periocular infections or active intraocular inflammation.
Apellis Pharmaceuticals is dedicated to helping patients access and receive support for Syfovre treatment. To this end, the company created "ApellisAssist," a program that offers Syfovre patients financial assistance (for eligible patients), disease education, insurance support, and ongoing product support. Patients and healthcare providers can obtain more information by calling 1-888-273-5547.
Currently, the European Medicines Agency is reviewing a marketing authorization application for Syfovre, with a decision anticipated in early 2024. Additionally, a marketing application for Syfovre has been submitted to Health Canada.
About SYFOVRE™ (pegcetacoplan injection)
SYFOVRE™ (pegcetacoplan injection) is the first and only approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration.