Penn’s complement inhibitor technology licensee, Apellis Pharmaceuticals, Inc. received U.S. Food and Drug Administration (FDA) approval for their drug SYFOVRE™ (intravitreal pegcetacoplan injection). SYFOVRE, which treats geographic atrophy (GA) secondary to age-related macular degeneration (AMD), is the first FDA-approved treatment for GA, a leading cause of blindness that impacts more than one million people in the U.S. and five million worldwide. Both SYFOVRE™ and EMPAVELI™ (pegcetacoplan), a prescription medicine used to treat paroxysmal nocturnal hemoglobinuria (PNH), are based on compstatin complement-inhibiting peptides discovered at Penn in the laboratory of John Lambris, Ph.D., the Dr. Ralph and Sallie Weaver Professor of Research Medicine in the Department of Pathology and Laboratory Medicine in the Perelman School of Medicine.
The FDA approval of SYFOVRE is based on positive results from the Phase 3 OAKS and DERBY studies conducted by Apellis for a period of 24 months across a broad and representative population of GA patients. In these studies, SYFOVRE was found to reduce the rate of GA lesion growth compared to sham and demonstrated increasing treatment effects over time, with the greatest benefit occurring between months 18-24.
Learn more on the complement inhibitor technology here and Apellis here.