Apellis' Syfovre Rejected Again in Europe for Geographic Atrophy
Key Insights
The European Medicines Agency reaffirmed its rejection of Apellis' Syfovre for treating geographic atrophy (search), despite the company's efforts to re-evaluate the supporting evidence.
This decision prevents Syfovre from entering the European market, where approximately 2.5 million individuals are affected by geographic atrophy (search), an irreversible cause of blindness.
The rejection was based on concerns that Syfovre did not demonstrate clinically meaningful benefits and carried significant risks of adverse events with regular injections.
Apellis Pharmaceuticals' Syfovre (pegcetacoplan) has been rejected again by European drug regulators for the treatment of geographic atrophy (search) (GA), despite the company's attempts to have the supporting evidence reevaluated. This decision effectively blocks the drug's availability in Europe, where an estimated 2.5 million people live with GA, a progressive form of vision loss.
EMA's Decision and Apellis' Response
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has upheld its earlier recommendation to reject the marketing authorization application for pegcetacoplan. Apellis CEO Cedric Francois expressed deep disappointment, stating that the decision leaves millions of Europeans without a treatment option for this irreversible condition. He noted the broad support for pegcetacoplan from the European retina community and dissenting votes from panel members who favored approval.
Reasons for Rejection
The EMA's decision was based on concerns that Syfovre did not provide clinically meaningful benefits to patients in clinical trials and that regular injections posed a significant risk of adverse events. This final rejection follows an initial negative opinion issued in January and a subsequent re-examination in June, both yielding the same outcome.
Syfovre's Approval and Challenges in the U.S.
In contrast to the European decision, Syfovre received approval in the United States in February 2023. The FDA's approval was based on the drug's ability to slow the growth of lesions associated with GA, potentially preserving eyesight. However, since its launch, Syfovre has faced challenges, including reports of rare but serious side effects, leading to a safety label update by the FDA.
Competitive Landscape and Future Plans
With the European market now out of reach, Apellis is restructuring its operations in the region, which includes laying off approximately 40 employees. The company is also awaiting regulatory decisions in the UK, Switzerland, Canada, and Australia. Meanwhile, Astellas Pharma's Izervay, a competitor to Syfovre, is under review for potential approval in Europe.
Analyst Perspectives
Mizuho Securities analyst Graig Suvannavejh noted that the outcome was not unexpected, given the low success rate of previous re-examination requests. He had previously estimated that Syfovre could generate approximately $367 million in peak yearly sales in Europe.