Apellis Pharmaceuticals, Inc. has announced it has submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) 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 five million people globally including one million people in the United States.
“Pegcetacoplan is the only treatment that has shown the potential to meaningfully slow disease progression across three large studies with a representative, real-world population of GA patients,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “With the submission of our marketing application in the United States, we are now one step closer towards our goal of bringing the first GA treatment to patients living with this relentless and irreversible disease.”
The NDA submission is based on results from the Phase 3 DERBY and OAKS studies at 12 and 18 months and the Phase 2 FILLY study at 12 months. In the studies, treatment with both monthly and every-other-month pegcetacoplan resulted in a clinically meaningful reduction of GA lesion growth across a broad, heterogenous population of more than 1,500 patients. Pegcetacoplan demonstrated a favorable safety profile in all three studies.
The FDA decision on NDA filing acceptance is expected in August 2022. Apellis also plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.
Read the full press release from Apellis.