The FDA approved pegcetacoplan injection (Syfovre) as the first treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), according to a statement from Apellis Pharmaceuticals.
Primary support for the approval came from the randomized, sham-controlled DERBY and OAKS trials, which showed a 22% overall reduction in growth of GA with pegcetacoplan over 24 months, with reductions of as much with 30% with monthly intravitreal injections.
“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, MD, PhD, of Duke University Medical Center in Durham, North Carolina, in the company statement. “Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined. With Syfovre, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”
Sometimes called “dry” or atrophic AMD, GA has an estimated prevalence of 1 million adults in the U.S. and 5 million worldwide. The progressive, vision-threatening condition evolves from cumulative retinal damage associated with aging, environmental stressors, and other factors. The damage causes inflammation affecting multiple regulatory pathways, including the complement cascade. Pegcetacoplan targets C3 protein, a key driver of the complement cascade.
DERBY and OAKS involved a total of 1,258 patients with confirmed GA secondary to AMD. They received monthly or bimonthly pegcetacoplan by intravitreal injection or sham injections. The primary endpoint was change in the total area of GA lesions at 12 months. Randomized treatment continued for 24 months.
The OAKS trial demonstrated superiority of pegcetacoplan at 12 and 24 months, whereas the DERBY trial did not show a statistically significant difference at 12 months. In a prespecified combined analysis of patients with extrafoveal lesions, monthly and bimonthly injections of pegcetacoplan reduced geographic atrophy by 26% and 23%, respectively, at 12 months (P<0.0001, P=0.0002).
The most common adverse events (≥5% of patients) associated with pegcetacoplan were ocular discomfort, neovascular AMD, vitreous floaters, and conjunctival hemorrhage.
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