Apellis grabs first FDA nod for geographic atrophy

Feb. 20, 2023

The U.S. FDA has approved Apellis Pharmaceuticals' injectable, Syfovre, making it the first and only approved treatment for geographic atrophy (GA), a leading cause of blindness.

GA is an advanced form of age-related macular degeneration. It is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. By targeting C3, Apellis' pegcetacoplan injection is designed to provide comprehensive control of the complement cascade, part of the body’s immune system.

The approval of Syfovre is based on positive results from the phase 3 OAKS and DERBY studies across a broad and representative population of patients. In the studies, the treatment 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 18-24.

“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, M.D., Ph.D., lead investigator for the OAKS study, director of ophthalmology clinical research, associate professor of ophthalmology. "Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined."

The Massachusetts-based biopharma expects to make Syfovre available by early March, through specialty distributors and specialty pharmacies nationwide. Additionally, a marketing authorization application is under review by the EMA with a decision expected in early 2024. A marketing application has been submitted to Health Canada.