Apellis announces FDA acceptance of NDA amendment and PDUFA date for pegcetacoplan for geographic atrophy

Apellis Pharmaceuticals Inc. today announced that the FDA has accepted the company’s unsolicited major amendment to the New Drug Application (NDA) for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

According to a news release, the updated Prescription Drug User Fee Act (PDUFA) goal date is February 26, 2023. The FDA also restated that they do not plan to hold an advisory committee meeting to discuss the application.

“With the inclusion of the 24-month data, we have the potential to have the best product profile at launch for pegcetacoplan, with minimal impact to launch timing,” said Cedric Francois, MD, PhD, CEO and co-founder, Apellis. “We appreciate the opportunity for the FDA to review these data and look forward to working with the agency to bring this first potential therapy to people living with GA as quickly as possible.”

Apellis announced earlier this month the company’s decision to submit 24-month efficacy data from the Phase 3 DERBY and OAKS studies as part of the NDA review. The 14-month data showed increasing and consistent effects with every-other-month and monthly pegcetacoplan treatment and a favorable safety profile in both studies.

According to the news release, Apellis remains on track to submit an EU marketing authorization application, which will also include the 24-month results, to the European Medicines Agency by the end of 2022.

About Pegcetacoplan for Geographic Atrophy
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.

About DERBY and OAKS
DERBY (n=621) and OAKS (n=637) are Phase 3, multicenter, randomized, 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.