FDA approves first treatment for geographic atrophy

Pegcetacoplan (SYFOVRE) has been approved by the U.S. Food and Drug Administration as the first and only treatment for geographic atrophy (GA), an advanced form of age-related macular degeneration.

Pegcetacoplan is an injection approved for GA patients with or without subfoveal involvement and allows a flexible dosing regimen of every 25 to 60 days.

The approval of pegcetacoplan was based on positive results from the OAKS (637 participants) and DERBY (621 participants) phase 3 studies. The OAKS and DERBY studies showed that compared with placebo, pegcetacoplan reduced the rate of GA lesion growth and demonstrated increasing treatment effects over time. Both monthly and every-other-month pegcetacoplan cut the rate of GA lesion growth through 24 months (OAKS: 22 percent monthly and 18 percent every other month; DERBY: 18 percent monthly and 17 percent every other month). The most common adverse reactions (incidence of ≥5 percent) were ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, and conjunctival hemorrhage.

"The approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade," lead investigator for the OAKS study, Eleonora Lad, M.D., Ph.D., of the Duke University Medical Center in Durham, North Carolina, said in a 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."

Approval of pegcetacoplan was granted to Apellis Pharmaceuticals.