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  • Potential Breakthrough Treatment for a Leading Cause of Blindness

    Promising results reported for what may be the first treatment for advanced form of age-related macular degeneration

    There is currently no treatment for geographic atrophy, the advanced “dry” form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. But that may change in the next couple of months. A new drug has shown it can slow the progression of this devastating eye condition. The researchers say the U.S. FDA could approve the new drug as early as late November of this year. The latest data was presented today at AAO 2022, the 126th annual meeting of the American Academy of Ophthalmology.

    Geographic atrophy (GA) affects the retina, a part of the eye that sends information to the brain to enable sight. GA refers to the area of the retina where cells waste away and die. These regions of atrophy result in a blind spot in the visual field. The new drug called pegcetacoplan is what’s known as a complement system inhibitor, meaning it aims to prevent this cell death from happening.  

    The study data presented today involved two phase three clinical trials (DERBY and OAKS) involving more than 1,200 patients with late-stage dry AMD. Patients were randomized into three groups: One received 15 milligrams of pegcetacoplan monthly; another received 15 milligrams every other month, and the third received no treatment at all. Patients included in the study included those with subfoveal and nonsubfoveal lesions.

    After one year of treatment, the area of atrophy reduced by 16 to 18 percent in patients treated every other month, and by 19 to 22 percent with monthly dosing.

    Even more important, the effect seemed to accelerate over the course of the 2-year study, especially between 18 months and two years, with reductions of 25 to 29 percent with every other month dosing, and by 24 to 36 percent with monthly dosing.

    “OAKS and DERBY represent the longest study of inhibition of the complement system to reduce the progression of geographic atrophy, said lead researcher,” Rishi P. Singh, MD. “The results are encouraging for both patients and physicians given the visual disability of this condition and the lack of effective treatments.”

    Though the results are promising, it’s important for people with geographic atrophy to understand that the new drug did not reverse vision loss. And it’s likely that if the new drug is approved in November, most people will need treatment for an extended period.

    About the American Academy of Ophthalmology

    The American Academy of Ophthalmology is the world’s largest association of eye physicians and surgeons. A global community of 32,000 medical doctors, we protect sight and empower lives by setting the standards for ophthalmic education and advocating for our patients and the public. We innovate to advance our profession and to ensure the delivery of the highest-quality eye care. Our EyeSmart® program provides the public with the most trusted information about eye health. For more information, visit