Potential Breakthrough Drug for Geographic Atrophy Awaiting FDA Approval

A new drug under accelerated review at the Food and Drug Administration (FDA) could slow the progression of vision loss from geographic atrophy, an advanced form of age-related macular degeneration (AMD). If approved, this would be the first-ever treatment in the U.S. for geographic atrophy, which affects an estimated 5 million people worldwide. A decision is expected in February 2023.

“A drug to slow the progression of this devastating disease would provide hope for millions of individuals affected by geographic atrophy,” said BrightFocus President and CEO Stacy Haller. “It is only through the tenacious work of the scientific community that these outcomes can be achieved.”

The drug, pegcetacoplan, was shown in clinical trials to slow the progression of geographic atrophy-caused vision loss.

In its 24-month results from two separate Phase 3 clinical studies, Apellis showed that the drug reduced geographic atrophy lesion growth compared to controls, and the treatment’s effect grew stronger between months 18-24. This accelerated treatment effect is promising and suggests that, over time, pegcetacoplan “is preserving more and more photoreceptor cells that are directly responsible for vision,” lead principal investigator for the OAKS study Eleonora Lad, MD, PhD (Duke) said in an Apellis press release.

In both clinical studies, pegcetacoplan’s safety profile was favorable.

In addition to being under FDA Priority Review with a Prescription Drug User Fee Act target action date in February 2023, Apellis said it will submit the 24-month results to the European Medicines Agency by the end of 2022.

[Read more: Clinical Trials: Your Questions Answered]

Learn More About Geographic Atrophy and Complement Inhibitors

Unlike another advanced form of macular degeneration, known as wet AMD, GA lesions remain dry but become undernourished and inflamed, creating dead zones (“atrophy”) of tissue in the central retina, including photoreceptors. This leads to blind spots in central vision, making it difficult to perform tasks like driving, reading, and recognizing faces. Eventually, if the lesions grow, GA causes central blindness.

Apellis and a handful of companies are targeting excessive activation of the complement system—our immune system’s early response to internal stressors—as a chief cause of GA. The so-called “complement cascade,” or overexpression of immune proteins, including complement C3 targeted by pegcetacoplan, leads to an unhealthy situation where chronic intraocular inflammation further stresses the retina, thinning it and the underlying tissue it depends on for nourishment, and ultimately causing cell death.

Learn more:

• What is Geographic Atrophy? (Expert Article)

• Geographic Atrophy Fact Sheet

• New Drugs Show Promise for Treating Geographic Atrophy (Research News)

• Apellis Announces 24-Month Phase 3 Post Hoc Results Showing Treatment with Pegcetacoplan Led to Preservation of Function in High-Risk Areas of the Retina in Patients with Geographic Atrophy (GA) at AAO Annual Meeting (Apellis press release)

About BrightFocus Foundation

BrightFocus Foundation is a premier nonprofit funder of research to defeat Alzheimer’s, macular degeneration, and glaucoma. Through its flagship research programs—Alzheimer’s Disease Research, National Glaucoma Research, and Macular Degeneration Research—the Foundation is currently supporting a $75 million portfolio of 287 scientific projects worldwide. BrightFocus has awarded nearly $275 million in groundbreaking medical research funding since inception and shares the latest research findings, expert information, and bilingual disease resources to empower the millions impacted by these devastating diseases. Join our community at brightfocus.org.