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Apellis Pharmaceuticals announce the approval of pegcetacoplan injection for the treatment of geographic atrophy (GA).

Apellis Pharmaceuticals, Inc. (NASDAQ: APLS) has announced that the U.S. Food and Drug Administration (FDA) has approved pegcetacoplan injection (labelled “SYFOVRE”), for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). According to the company, pegcetacoplan injection is the “first and only FDA-approved treatment for GA”, potentially impacting more than one million people in the U.S., and five million people worldwide”.  Pegcetacoplan  is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to complement C3 and C3b.  As well known, the complement system plays critical roles in both innate and adaptive immunity, contributing to immune surveillance, inflammation, and homeostasis via three different activationpathways (classical, alternative, and lectin) and numerous effector molecules.  The diverse functions of the peptide include recruitment and activation of immune cells, opsonization of pathogens to target them for phagocytosis, and direct destruction of pathogens by the membrane attack complex.


The approval of pegcetacoplan was based on positive results from the Phase 3 “OAKS” and “DERBY” studies at 24 months across a broad and representative population of patients. The studies of OAKS (NCT NCT03525613, with a n=637) and DERBY (NCT NCT03525600; n=621) were multi-center, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated the efficacy of monthly and every-other-month in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 24 months.  Following the approval, Jeff Todd, President and CEO of Prevent Blindness commented that, “[f]]or the first time ever, we are celebrating the approval of a treatment for GA. This is a historic and hopeful day for all GA patients and their care partners, who have been waiting for a treatment for this relentless form of vision loss.”


Pegcetacoplan is expected to be available by the beginning of March through specialty distributors and specialty pharmacies nationwide. A marketing authorization application 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.  Following the clinical research achievement, Eleonora Lad, M.D., Ph.D., lead investigator for the OAKS study, and associate professor of ophthalmology, Duke University Medical Center said, “[t]he approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade.  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.”