A Complement Inhibitor for Dry Macular Degeneration

The April 3 issue of The Medical Letter includes an article on the first treatment ever approved by the FDA for use in patients with dry age-related macular degeneration (AMD). More specifically, pegcetacoplan (Syfovre) was approved for treatment of geographic atrophy, which is a late stage of dry AMD.

AMD is common; according to the CDC, the prevalence increases from about 2% among people 40-44 years old to 47% among those more than 85 years old. Presently it affects about 200 million people worldwide (about 20 million in the US) and is the most common cause of blindness in developed countries. About 90% of patients have dry AMD, and 10% develop wet (neovascular) AMD. Progression of dry AMD can lead to wet AMD, geographic atrophy, or both.

Wet AMD, characterized by fluid accumulating from leaky new blood vessels, can cause severe vision loss, but in recent years it has become treatable with VEGF (vascular endothelial growth factor) inhibitors; injected into the eye every 1-3 months, these drugs are highly effective at removing the fluid and maintaining vision. Started soon after symptoms first appear, they can restore a substantial percentage of vision lost up to that point.

Dry AMD progresses much more slowly, but when geographic atrophy develops, it can also lead to blindness. A dietary supplement of antioxidant vitamins and minerals (AREDS 2) is widely recommended to prevent or delay progression to advanced AMD, but it has not been approved for such use by the FDA, and its efficacy has been modest at best (Nutritional supplements for age-related macular degeneration revisited. Med Lett Drugs Ther 2013; 55:50).

The complement cascade has been linked to the development of dry AMD and geographic atrophy (D Desai and PU Dugel. Complement cascade inhibition in geographic atrophy: a review. Eye 2022; 36:294). Pegcetacoplan is the first of a number of complement inhibitors that have been studied for treatment of this disorder. Atrophy is not reversible, of course, but the hope is that these drugs, which are injected periodically into the affected eye(s), will prevent or at least reduce its progression.

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