FDA Accepts NDA for Pegcetacoplan for Advanced Macular Degeneration

Pegcetacoplan is a targeted therapy to treat patients with age-related macular degeneration. The Prescription Drug User Fee Act (PDUFA) target action date is Nov. 26, 2022.

The FDA has accepted and granted priority review designation for the new drug application (NDA) for pegcetacoplan. Developed by Apellis Pharmaceuticals, pegcetacoplan is an investigational, targeted C3 therapy for the treatment of patients with geographic atrophy secondary to age-related macular degeneration (AMD). The therapy is designed to be injected into the back of the eye. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date is Nov. 26, 2022.

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.

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, heterogeneous population of more than 1,500 patients. Pegcetacoplan demonstrated a favorable safety profile in all three studies.

The DERBY and OAKS studies were presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in May 2022 in Denver. At 18 months, monthly and every-other-month treatment with pegcetacoplan showed a continuous and clinically meaningful reduction in the growth of lesions within the fovea, which is located in the center of the macula, as well as lesions outside of the fovea. In the combined studies, pegcetacoplan reduced extrafoveal geographic atrophy lesion growth by 26% for monthly treatment and 21% with every-other-month treatment. At 18 months, foveal lesion growth was reduced 13% for both monthly and every-other-month treatment.

Apellis had submitted its application in June 2022.