FDA approves Apellis' pegcetacoplan for geographic atrophy therapy

The FDA has approved intravitreal pegcetacoplan (SYFOVRE; Apellis) to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The approval is based on the results of the 24-month phase 3 data submitted in its New Drug Application in November 2022.

“The approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, MD, PhD, lead investigator for the OAKS study, director of ophthalmology clinical research, associate professor of ophthalmology, Duke University Medical Center, in a prepared statement. “Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined. With SYFOVRE, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”

Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of GA.

Inclusion of the 24-month data, the company explained, strengthened the product profile at launch, with minimal impact to launch timing, and is considered a major amendment to the NDA and extended the review period by 3 months.

The 24-month efficacy data were obtained from the Phase 3 DERBY and OAKS studies. According to a press release, “These data showed robust and consistent effects with monthly and every-other-month pegcetacoplan treatment in both studies. The 24-month safety data were previously submitted as part of the 120-day update.”

Apellis noted that the 24-month phase 3 data “demonstrated robust and increasing effects over time.” The longer-term data provided an even stronger product profile at launch,” according to Cedric Francois, MD, PhD, chief executive officer and co-founder, Apellis.

The OAKS (n = 621) and DERBY (n = 637) are Phase 3, multicenter, randomised, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies was to evaluate the efficacy of monthly and every-other-month pegcetacoplan 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.

Francois continued, “We will work closely with the FDA as they complete their review of the pegcetacoplan NDA and look forward to bringing the first potential therapy to people living with GA, who currently have no approved treatment for this relentless and debilitating disease.”

A marketing authorisation application for pegcetacoplan is under review by the European Medicines Agency with a decision expected in early 2024. In addition, a marketing application has been submitted to Health Canada.