Apellis submits marketing authorisation application to the European Medicines Agency for pegcetacoplan for geographic atrophy

Apellis Pharmaceuticals Inc. announced that the company has submitted a marketing authorisation application (MAA) to the European Medicines Agency for intravitreal pegcetacoplan, an investigational, targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

GA is a leading cause of blindness that impacts more than 5 million people globally.1,2

“Along with the upcoming FDA decision in the U.S., the submission of our EU marketing application brings us closer than ever toward our goal of delivering pegcetacoplan to patients living with GA around the world,” said Jeffrey Eisele, PhD, chief development officer at Apellis. “If approved, pegcetacoplan would be the first and only treatment for GA in Europe.”

Frank G. Holz, DERBY and OAKS study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany, pointed out that pegcetacoplan is the only GA treatment to demonstrate increased effects over 24 months across a broad patient population.

“There are currently no treatments to slow the progression of GA, a life-altering disease that causes substantial vision loss, so the potential approval of pegcetacoplan would be a significant milestone for patients in Europe,” he said in a statement.

The MAA submission is based on results from the Phase 3 DERBY and OAKS studies at 24 months. In the studies, treatment with both every-other-month and monthly pegcetacoplan demonstrated robust and clinically meaningful reductions of GA lesion growth, with treatment effects that increased over time, and a favourable safety profile.

The U.S. marketing application is under review with a Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2023.

About DERBY and OAKS
DERBY (n=621) and OAKS (n=637) are Phase 3, multicenter, randomised, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (p-value less than .05) at 12 months. Patients continued to receive masked treatment for 24 months.