The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for pegcetacoplan for the treatment of geographic atrophy (GA).

Pegcetacoplan is an intravitreal targeted C3 therapy that works by regulating excessive activation of the complement cascade. The NDA submission is supported by data from the phase 3 DERBY (ClinicalTrials.gov Identifier: NCT03525600) and OAKS (ClinicalTrials.gov Identifier: NCT03525613) studies, and the phase 2 FILLY study (ClinicalTrials.gov Identifier: NCT02503332).

The phase 3, randomized, double-masked, DERBY (N=621) and OAKS (N=637) studies evaluated the efficacy and safety of intravitreal pegcetacoplan vs sham injections in patients with GA. The primary endpoint for both studies was the change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 12 months. The trials also included an extension phase, where patients continued to receive masked treatment for up to 24 months. Results showed that both monthly and every-other-month treatment slowed GA disease progression when compared with sham. 

The phase 2, randomized, single-masked, sham-controlled FILLY study (N=246) evaluated the efficacy and safety of intravitreal pegcetacoplan in patients with GA secondary to age-related macular degeneration (AMD). Findings demonstrated that treatment with pegcetacoplan reduced the progression of GA secondary to AMD (as assessed by change in square root GA lesion size from baseline measured by fundus autofluorescence) at month 12.

Pegcetacoplan demonstrated a favorable safety profile in all 3 studies consistent with long-term intravitreal injection exposure.

“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. “Both monthly and every-other-month pegcetacoplan meaningfully slowed GA disease progression across 3 large studies, and the potential approval of this medicine would be a breakthrough for patients.”

A Prescription Drug User Fee Act (PDUFA) target date of November 26, 2022 has been set for the application.

References

  1. Apellis announces FDA acceptance and Priority Review of the New Drug Application for pegcetacoplan for the treatment of geographic atrophy (GA). News release. Apellis Pharmaceuticals, Inc. Accessed July 19, 2022. https://www.globenewswire.com/news-release/2022/07/19/2481617/0/en/Apellis-Announces-FDA-Acceptance-and-Priority-Review-of-the-New-Drug-Application-for-Pegcetacoplan-for-the-Treatment-of-Geographic-Atrophy-GA.html
  2. Apellis announces detailed 18-month results from phase 3 DERBY and OAKS studies of pegcetacoplan for geographic atrophy (GA) at ARVO Annual Meeting. News release. Apellis Pharmaceuticals, Inc. May 2, 2022. Accessed July 19, 2022. https://investors.apellis.com/news-releases/news-release-details/apellis-announces-detailed-18-month-results-phase-3-derby-and