FDA grants Priority Review for intravitreal pegcetacoplan BLA from Apellis

Apellis Pharmaceuticals Inc. today announced that the FDA has accepted and granted Priority Review designation for the intravitreal pegcetacoplan New Drug Application (NDA).

According to the company, pegcetacoplan is an investigational, targeted C3 therapy for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

The company noted in its release that the Prescription Drug User Fee Act (PDUFA) target action date is November 26, 2022. The FDA indicated that it is not currently planning to hold an advisory committee meeting to discuss the application.

“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,” Charles Wykoff, MD., PhD, an OAKS study investigator and director of research, Retina Consultants of Texas, said in the news release. “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.”

Apellis noted in its release that 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.

Acording to the company, 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 1500 patients. Pegcetacoplan demonstrated a favorable safety profile in all three studies.

Jeffrey Eisele, PhD, chief development officer at Apellis, noted that the milestone marks the company’s second NDA acceptance in less than 2 years, a testament to the potential of targeting C3 to treat serious diseases with significant unmet need.

“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,” Eisele noted in the news release.

Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. Pegcetacoplan was previously granted Fast Track designation by the FDA for the treatment of GA secondary to AMD.

Apellis plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.

About DERBY and OAKS

DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomly assigned, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

According to the company, patients in DERBY and OAKS will continue to receive masked treatment for 24 months. Secondary functional endpoints will be evaluated after all patients have received treatment for 24 months.

About FILLY

The FILLY study was a 246-patient, Phase 2, multicenter, randomly assigned, single-masked, sham-controlled study evaluating the efficacy and safety of intravitreal pegcetacoplan in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).