The NDA was granted Priority Review designation with a PDUFA target action date of November 26, 2022.
The US Food and Drug Administration (FDA) has accepted and granted Priority Review designation for the intravitreal pegcetacoplan New Drug Application (NDA) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
The Prescription Drug User Fee Act (PDUFA) target action date is November 26, 2022, according to a release from Apellis Pharmaceuticals, Inc. Currently, the FDA is not planning to hold an advisory committee meeting to discuss the application for the investigational, targeted C3 therapy.
“This is a very exciting time for the retina community as we are closer than ever to having the first treatment for GA, an irreversible and devastating disease that causes a tremendous amount of vision loss,” said Charles Wykoff, MD, PhD, OAKS study investigator and director of research, Retina Consultants of Texas in the release.
This NDA submission was based on results from the Phase 3 DERBY and OAKS studies at 12 and 18 months, as well as the Phase 2 FILLY study at 12 months.
In DERBY and OAKS, the primary objective was to evaluate the efficacy of pegcetacoplan with sham injections 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 continued to receive masked treatment for 24 months.
The FILLY study evaluated 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 following treatment.
In each of the studies, the investigators found treatment with both monthly and every-other-month pegcetacoplan was linked to a clinically meaningful reduction of GA lesion growth across a broad, heterogeneous population of ≥1,500 patients. The therapy additionally showed a favorable safety profile in all three studies, the studies reported.
“Both monthly and every-other-month pegcetacoplan meaningfully slowed GA disease progression across three large studies, and the potential approval of this medicine would be a breakthrough for patients,” Wycoff added.
Previously, pegcetacoplan was granted Fast Track designation by the FDA for the treatment of GA secondary to AMD.
“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,” said Jeffrey Eisele, PhD, chief development officer at Apellis in the release. “We are committed to working closely with the FDA to bring this potential, first-ever treatment to patients living with GA as quickly as possible.”