CHMP Upholds Negative Opinion for Apellis' Pegcetacoplan for GA in the EU Following Re-Examination

Apellis Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has upheld its previous June 2024 negative opinion on the marketing authorization application for geographic atrophy (GA) treatment pegcetacoplan.

“We are deeply disappointed by this outcome, which leaves millions of Europeans with GA without a treatment for this irreversible form of blindness,” Cedric Francois, MD, PhD, CEO and co-founder of Apellis, said in a company news release. “This CHMP opinion was made despite broad support for pegcetacoplan from the European retina community and multiple dissenting votes by CHMP members who supported a path to approval.”

Apellis said it remains committed to advancing the treatment in other regions. “We have seen the profound impact this medicine has had for GA patients in the US and remain focused on expanding access to this crucial treatment, prioritizing unmet patient needs globally,” Dr. Francois said.

This marks the third rejection this year for pegcetacoplan in Europe, where there are currently no approved therapies for GA. The CHMP initially issued a negative opinion on the marketing authorization application (MAA) in January. The FDA approved pegcetacoplan for GA under the brand name Syfovre in February of 2023, making it the first approved therapy in the US for GA. 

“It is disheartening to know that we will not be able to provide access to this treatment to GA patients in the EU," said Professor Frank G. Holz, who served as an investigator for Apellis’ OAKS and DERBY studies. “As a specialist, I’ve seen firsthand how patients with GA lose their ability to read, drive, and even recognize faces. The phase 3 data for pegcetacoplan were clinically meaningful and demonstrated real potential for EU patients.”