The company noted that pegcetacoplan, 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, was granted Fast Track designation by the FDA for the treatment of geographic atrophy.
Apellis Pharmaceuticals Inc. today announced it has submitted a New Drug Application (NDA) to the 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 5 million people globally including 1 million people in the United States.1,2
According to the company, pegcetacoplan is 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. It was granted Fast Track designation by the FDA for the treatment of geographic atrophy.
“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,” Jeffrey Eisele, PhD, chief development officer at Apellis, said in a news release. “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.”
According to the company, 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 company noted in its news release that DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomly assigned, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in 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 at 12 months. Patients in DERBY and OAKS will continue to receive masked treatment for 24 months. Secondary functional endpoints will be evaluated after all patients have received treatment for 24 months.
The company noted that its FILLY study was a 246-patient, Phase 2, multicenter, randomly assigned, single-masked, sham-controlled study evaluating the efficacy and safety of intravitreal pegcetacoplan in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the study was to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in square root GA lesion size from baseline as measured by fundus autofluorescence at 12 months followed by six months of monitoring after the end of treatment.
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.
1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580.
2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.
3 Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.
4 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.