FDA Updates Target Date for Pegcetacoplan

Appellis had submitted updated data for the pegcetacoplan in age-related macular degeneration, which is considered a major amendment to the NDA. The new PDUFA date is Feb. 26, 2023.

The FDA has accepted Apellis Pharmaceuticals’ major amendment to the new drug application (NDA) for intravitreal pegcetacoplan to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The updated Prescription Drug User Fee Act (PDUFA) goal date is Feb. 26, 2023.

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. It is designed to be injected into the back of the eye. Geographic atrophy is an advanced form of age-related macular degeneration and a leading cause of blindness that impacts more than 5 million people worldwide, including 1 million people in the United States. There are currently no treatments for geographic AMD.

Related: FDA Accepts NDA for Pegcetacoplan for Advanced Macular Degeneration

Apellis had submitted to the FDA 24-month efficacy data from the phase 3 DERBY (which enrolled 637 patients) and OAKS (which enrolled 621 patients) 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. Both studies showed clinically meaningful reduction in geographic atrophy lesion growth from baseline compared with sham. Geographic atrophy lesion growth is correlated with loss of visual function over longer periods of time

Between months 18 and 24, the pegcetacoplan treatment effect accelerated compared with previous six-month periods, with robust reductions of geographic atrophy lesion growth versus sham. The increased effects were driven by a greater slowing of lesion growth by pegcetacoplan and not by an increase in the lesion growth rate in the sham group,

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.