FDA Approves First and Only Treatment for Geographic Atrophy, an Advanced Form of Age-Related Macular Degeneration

Global vision and brain research non-profit BrightFocus Foundation celebrates the U.S. Food and Drug Administration’s (FDA) approval of Syfovre (pegcetacoplan injection), the first-ever treatment to slow the progression of vision loss from geographic atrophy, an advanced form of dry age-related macular degeneration and a leading cause of blindness.

“Today’s FDA approval of Syfovre provides hope to the more than five million people worldwide who are at risk of permanent vision loss from geographic atrophy,” said BrightFocus President and CEO Stacy Pagos Haller. “This first-of-its-kind treatment is a momentous step forward in vision research and will make a meaningful difference in the lives of millions of people.”

An estimated one million people in the U.S. have geographic atrophy, an advanced and severe form of age-related macular degeneration in which regions of cells in the retina waste away and die (atrophy). This progressive and irreversible disease can lead to permanent vision loss. Nearly 20 million adults in the U.S. have some form of age-related macular degeneration (AMD), almost double the previous estimate of 11 million people, according to a new report.

People living with geographic atrophy often experience emotional hardships including anxiety, feeling powerless, and frustration. Approximately one in three have recently withdrawn from their social lives because of their disease, and BrightFocus offers a free AMD community group and educational audio chats for affected individuals.

How does it work?

Syfovre was approved in an accelerated FDA process used for treatments of serious or life-threatening illnesses that provide a meaningful therapeutic advantage over existing treatments. In clinical trials, Syfovre was shown to reduce the rate of geographic atrophy lesion growth by up to 36% with monthly injection. It works by targeting a specific protein in the complement pathway, a part of the immune system.

Syfovre is approved for all patients with geographic atrophy, with dosing flexibility every 25 to 60 days.

Earlier studies funded by the BrightFocus Macular Degeneration Research (MDR) program have contributed to scientists’ understanding of the role of complements in age-related macular degeneration pathobiology. A recent MDR-funded project examined how to determine the molecular factors responsible for age-related macular degeneration pathology and use that knowledge to develop complement-based gene therapy.

What’s next?

Syfovre is expected to be available by the beginning of March through select 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.

Read the press release from Apellis and learn more about geographic atrophy.