Apellis Denied CHMP Approval for Pegcetacoplan for GA in EU After Re-Examination

On September 20, 2024, Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) declared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has reaffirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for treating geographic atrophy (GA) secondary to age-related macular degeneration.

Cedric Francois, M.D., Ph.D., who serves as the chief executive officer and co-founder of Apellis, expressed deep disappointment regarding the decision. This outcome leaves millions of Europeans suffering from GA without a treatment for this irreversible form of blindness. Despite receiving extensive support from the European retina community and several dissenting votes from CHMP members advocating for the drug's approval, the negative opinion remained unchanged.

Dr. Francois emphasized the significant impact pegcetacoplan has had on GA patients in the United States. He remains committed to expanding access to this vital treatment, focusing on addressing the unmet needs of GA patients in the U.S. and other regions worldwide.

Frank G. Holz, a study investigator for OAKS and DERBY and professor and chairman of the department of ophthalmology at the University of Bonn, Germany, also expressed his disappointment. As an ophthalmologist and retina specialist, he has witnessed firsthand the debilitating effects of GA, which robs patients of their ability to read, drive, and even recognize faces. He noted that the Phase 3 data for pegcetacoplan are clinically significant and have the potential to make a difference for patients in the EU.

Geographic atrophy (GA) is an advanced form of age-related macular degeneration and one of the leading causes of blindness globally. It affects over one million individuals in the United States and five million people worldwide. GA is a progressive, irreversible disease characterized by the development of lesions that destroy retinal cells essential for vision. The vision loss caused by GA significantly impairs independence and quality of life, making everyday activities challenging. Typically, it takes approximately 2.5 years for GA lesions to begin affecting the fovea, which is responsible for central vision.

Pegcetacoplan is an investigational therapy targeting the C3 protein and is designed to modulate the excessive activation of the complement cascade, a component of the body’s immune system. This excessive activation can lead to the onset and progression of numerous serious diseases. SYFOVRE® (pegcetacoplan injection) is approved in the United States for the treatment of GA secondary to age-related macular degeneration.

Apellis Pharmaceuticals, Inc., is a global biopharmaceutical company committed to developing transformative therapies for some of the most challenging diseases patients face. The company pioneered a new class of complement medicine in 15 years and now has two approved medicines that target C3, including the first-ever therapy for geographic atrophy, a major cause of blindness worldwide. Apellis believes it has only begun to explore the potential of targeting C3 across various serious retinal, rare, and neurological diseases.