Apellis Announces Negative CHMP Opinion for Pegcetacoplan for GA in the European Union and Plans to Seek Re-Examination of Application

WALTHAM, Mass., Jan. 26, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). As previously announced, this opinion was expected based on a negative trend vote following an oral explanation meeting in December 2023. Apellis plans to seek immediate re-examination of its application.

“We are very disappointed in this outcome on behalf of the millions of Europeans with GA who currently have no treatment options for this irreversible form of blindness,” said Jeffrey Eisele, Ph.D., chief development officer, Apellis. “We plan to promptly initiate the re-examination of our application and address CHMP questions, with the goal of bringing this first-ever potential treatment to the GA community in Europe.”

The MAA is based on results from the Phase 3 OAKS and DERBY studies at 24 months, which were published in The Lancet. Treatment with both every-other-month and monthly pegcetacoplan reduced GA lesion growth with increasing treatment effects over time and showed a well-demonstrated safety profile. Additionally, pegcetacoplan preserved visual function longer in multiple post hoc Phase 3 analyses that have been presented at medical congresses.

More than 2.5 million people in Europe are living with GA,¹ a progressive and irreversible form of vision loss that severely impairs independence and quality of life.

About the Phase 3 OAKS and DERBY Studies
OAKS (n=637) and DERBY (n=621) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections across a broad and heterogenous population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The studies evaluated 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.