Apellis to Seek Re-Examination After Negative CHMP Opinion for Pegcetacoplan in EU

On June 28, 2024, Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) disclosed that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on their marketing authorization application for intravitreal pegcetacoplan aimed at treating geographic atrophy (GA).

Despite this setback, the decision was not unanimous; several CHMP members dissented from the negative opinion. Apellis intends to seek a re-examination and expects a conclusive opinion by the fourth quarter of 2024. The recent opinion followed an Ad Hoc Expert Group meeting, which concluded that the size of GA lesions is an appropriate primary outcome measure for trials in GA, and microperimetry is the most effective functional measure.

The review process was managed by the original rapporteurs after the EMA reset to Day 180 of the initial marketing authorization assessment.

Jeffrey Eisele, Ph.D., chief development officer at Apellis, stated, "We remain committed to providing a treatment option for GA patients in Europe, who currently lack effective therapies for this debilitating disease leading to irreversible vision loss." He emphasized the European retina community's broad support for pegcetacoplan and the company's focus on swiftly initiating the re-examination process to deliver this crucial treatment.

Geographic atrophy (GA) represents an advanced stage of age-related macular degeneration and stands as a major cause of blindness globally. GA affects over one million Americans and five million individuals worldwide. The disease is marked by the progressive and irreversible expansion of lesions that degrade retinal cells necessary for vision. Vision impairment from GA significantly hampers independence and quality of life, making everyday activities challenging. Typically, it takes approximately 2.5 years for GA lesions to affect the fovea, which is crucial for central vision.

Pegcetacoplan is an investigational therapy aimed at regulating excessive activation of the complement cascade, part of the immune system that, when activated excessively, can lead to the progression of various severe diseases. SYFOVRE® (pegcetacoplan injection) is already approved in the United States for treating GA secondary to age-related macular degeneration.

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company dedicated to developing transformative therapies for some of the most complex diseases. The company has pioneered the first new class of complement medicine in 15 years, with two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness worldwide. Apellis believes it has just begun to explore the potential of targeting C3 in treating serious retinal, rare, and neurological diseases.