Apellis faces setback in EU for pegcetacoplan approval on Sept 20

On September 20, 2024, Apellis Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) upheld its negative opinion regarding the marketing authorization application for pegcetacoplan, intended for treating geographic atrophy (GA) linked to age-related macular degeneration. This decision follows a previous negative opinion issued in June 2024, which has left many patients in Europe without viable treatment options for this debilitating condition. The CEO of Apellis, Cedric Francois, expressed deep disappointment over the CHMP's decision, emphasizing the lack of treatment alternatives for millions suffering from GA. Despite the negative ruling, there was significant support for pegcetacoplan from the European retina community, with several CHMP members advocating for its approval. This dissent highlights the ongoing debate within the medical community regarding the potential benefits of the drug. Frank G. Holz, a prominent ophthalmologist involved in the OAKS and DERBY studies, also voiced his disappointment, noting the severe impact GA has on patients' daily lives, including their ability to read and drive. He pointed out that the Phase 3 clinical data for pegcetacoplan demonstrated meaningful clinical outcomes, suggesting that the drug could significantly improve the quality of life for patients in the EU. Geographic atrophy is a progressive and irreversible disease that leads to vision loss, affecting millions globally. The failure to secure approval for pegcetacoplan underscores the challenges faced by pharmaceutical companies in navigating regulatory processes, particularly for conditions that lack effective treatments.