Apellis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted Apellis' 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). 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.
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 24-month data showed increasing and consistent effects with every-other-month and monthly pegcetacoplan treatment and a favorable safety profile in both studies. 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. 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 0.05) at 12 months. Patients continued to receive masked treatment for 24 months.