Apellis’ Treatment for Advanced Dry AMD to Receive Priority Review from FDA

Apellis Pharmaceuticals has announced that the US Food & Drug Administration (FDA) has accepted its new drug application (NDA) for pegcetacoplan, the company’s emerging therapy for geographic atrophy (GA), the advanced form of dry age-related macular degeneration. If the NDA is approved, Apellis would be able to market the drug in the US.

The FDA granted priority review of the pegecetacoplan NDA with a target action date of November 26, 2022. Priority review reduces the time the FDA takes to evaluate the NDA from 10 months to 6 months.

Apellis plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.

Pegcetacoplan was evaluated for GA in two Phase 3 clinical trials, DERBY and OAKS, each of which enrolled more than 600 patients. In the combined trials at 18 months, pegcetacoplan reduced extrafoveal GA lesion growth by 26 percent and 21 percent with monthly and every-other-month treatment, respectively. Pegcetacoplan reduced foveal lesion growth by 13% for those receiving monthly or every-other-month injections. The treatment was administered by injections into the vitreous, the soft gel in the middle of the eye.

GA lesions are the regions in the retina where degeneration from dry AMD has occurred. The fovea is a small pit in the center of the retina, rich in cone photoreceptors, that provides the sharpest vision, including the ability to read and recognize faces. GA lesions often first develop outside of the fovea — these are known as extrafoveal lesions. Over time, the lesions encroach on the foveal region leading to more significant vision loss. Experts believe that targeting extrafoveal lesions with treatments before they affect the fovea may be the optimal strategy for preserving vision in people with GA.

“We are very encouraged by the FDA’s priority review for Apellis’ emerging therapy for geographic atrophy,” said Claire Gelfman, PhD, chief scientific officer at the Foundation Fighting Blindness. “The condition is a major unmet medical need leading to significant vision loss for at least one million people over 55 in the US and millions more around the world.”