Apellis Pharmaceuticals announced on February 17, 2023, that the US Food and Drug

 Administration (FDA) has approved Syfovre (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Syfovre is the first and only FDA-approved treatment for GA, a leading cause of blindness that impacts more than 1 million people in the United States and 5 million people worldwide.^1,2

“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 and director of ophthalmology clinical research at 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.”

The approval of Syfovre is based on positive results from the phase 3 OAKS (n=637) and DERBY (n=621) studies at 24 months across a broad and representative population of patients. Syfovre is 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. The safety profile of Syfovre is well demonstrated following approximately 12,000 injections. The most common adverse reactions (≥5%) reported in patients receiving SYFOVRE were ocular discomfort, neovascular AMD, vitreous floaters, and conjunctival hemorrhage.

In the OAKS and DERBY studies, Syfovre 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 and 24. Both monthly and every-other-month Syfovre reduced the rate of GA lesion growth through 24 months compared to sham:

• OAKS: 22% monthly; 18% every-other-month
• DERBY: 18% monthly; 17% every-other-month

Syfovre is expected to be available by the beginning of March through specialty distributors and specialty pharmacies nationwide. A marketing authorization application for Syfovre is under review by the European Medicines Agency, with a decision expected in early 2024. In addition, a marketing application has been submitted to Health Canada. Apellis has also launched the ApellisAssist program designed to help Syfovre patients along their treatment journey by providing a system inclusive of insurance support, financial assistance for eligible patients, disease education, and ongoing product support.

References

1. Rudnicka AR, Jarrar Z, Wormald R, Cook DG, Fletcher A, Owen CG. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology. 2012;119(3):571-580. doi:10.1016/j.ophtha.2011.09.027
2. Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health. 2014;2(2):e106-e116. doi:10.1016/S2214-109X(13)70145-1