Apellis' Syfovre Faces Third Rejection by EU's CHMP for Geographic Atrophy
核心洞察
Apellis Pharmaceuticals' Syfovre (pegcetacoplan) received a third negative opinion from the CHMP for treating geographic atrophy (搜索) (GA) in the EU.
The decision follows previous rejections and a procedural reset due to a European Court of Justice ruling on EMA's expert group organization.
Syfovre is approved in the U.S. for GA, demonstrating strong initial uptake with $292.1 million in sales in the first half of 2024.
Apellis Pharmaceuticals' shares fell 11.5% after the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) confirmed its negative opinion on the marketing authorization application (MAA) for intravitreal pegcetacoplan (Syfovre) for the treatment of geographic atrophy (搜索) (GA) secondary to age-related macular degeneration (搜索) (AMD). This marks the third rejection by the CHMP for this indication.
The company expressed disappointment, highlighting the lack of available treatments for GA patients in the EU. Apellis noted that the CHMP's decision was made despite support from the European retina community and dissenting votes from some CHMP members.
Regulatory Hurdles and Procedural Reset
The initial CHMP rejection occurred in January 2024, prompting Apellis to request a re-examination. Subsequently, the Court of Justice of the European Union (CJEU) issued a judgment impacting EMA's expert group policies, leading to a procedural reset of the Syfovre MAA review in April 2024. Apellis clarified that this reset was solely due to the CJEU judgment and not related to any deficiencies in the drug submission package.
Despite these efforts, the CHMP issued a second negative opinion in June 2024, which has now been confirmed.
Syfovre's Success in the U.S.
In contrast to the EU situation, Syfovre received FDA approval in the United States in February 2023 as the first and only treatment for GA secondary to AMD. GA affects over one million people in the U.S. and five million worldwide, making it a leading cause of blindness. Syfovre has demonstrated strong initial uptake in the U.S. market, with sales reaching $292.1 million in the first half of 2024, a 241% increase year-over-year. As of June 30, 2024, approximately 330,000 doses have been delivered since its launch.
Ongoing Efforts and Other Indications
Apellis is pursuing marketing authorization for intravitreal pegcetacoplan in other countries, with decisions expected later this year. The company also markets pegcetacoplan under the brand names Empaveli/Aspaveli in the U.S. and EU for the treatment of paroxysmal nocturnal hemoglobinuria (搜索).