Apellis receives FDA acceptance, priority review of NDA for GA treatment

Apellis Pharmaceuticals announced Tuesday that the FDA has accepted and granted it priority review designation for the New Drug Application (NDA) of intravitreal pegcetacoplan.

As an investigational, targeted C3 therapy, pegcetacoplan is used for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). It was previously granted Fast Track designation by the FDA in 2018.

The latest NDA submission is based on results from the DERBY and OAKS studies (both phase 3) at 12 and 18 months as well as the FILLY study (phase 2) at 12 months, according to a company news release.

Further, treatment with both monthly and every-other-month pegcetacoplan injections resulted in a clinically meaningful reduction of GA lesion growth in all three studies’ broad, heterogeneous population of 1,500 patients.

“This important milestone marks our second NDA acceptance in less than two years, a testament to the potential of targeting C3 to treat serious diseases with significant unmet need,” stated Jeffrey Eisele, PhD, Apellis’s chief development officer, in the release.

The PDUFA target action date for pegcetacoplan is November 26, 2022. If approved, pegcetacoplan will be the first-ever treatment for GA.