The FDA has accepted Apellis Pharmaceuticals’ amendment to change its new drug application (NDA) for pegcetacoplan (Syfovre) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
First off, what is pegcetacoplan?
Pegcetacoplan is an investigational targeted C3 therapy that works by regulating excessive activation of the complement cascade, which can lead to the onset and progression of diseases. The FDA previously granted it a Fast Track designation for GA treatment.
Tell us about the studies.
The DERBY and OAKS studies compared pegcetacoplan’s efficacy and safety with the use of sham injections. Investigators tracked the efficacy of monthly and bimonthly pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline measured by fundus autofluorescence at 12 months. Patients continued to receive masked treatment for 24 months. (via)
Both monthly and bimonthly injections showed a clinically meaningful reduction—36% in DERBY and 29% in OAKS—in GA lesion growth when compared to the sham injections from months 18 to 24.
So when’s this new PDUFA date?
The goal date is now February 26, 2023. Along with this update, the FDA reiterated that they will not have an advisory committee meeting to discuss the NDA application.
What’s the significance of this?
If approved, pegcetacoplan would be the first FDA-approved treatment therapy for patients with GA.