Astellas has announced the withdrawal of its marketing authorization application to the European Medicines Agency (EMA) for avacincaptad pegol, a treatment for geographic atrophy (GA). This decision follows interactions with the EMA's Committee for Medicinal Products for Human Use (CHMP), where concerns were raised regarding the drug's benefit-risk profile. The withdrawal represents a significant setback for Astellas, particularly after its $5.9 billion acquisition of Iveric Bio, the original developer of avacincaptad pegol.
Regulatory Hurdles in Europe
While avacincaptad pegol has secured approval in the United States under the brand name Izervay for GA secondary to age-related macular degeneration (AMD), the path to European approval has proven challenging. The CHMP's specific reservations have not been publicly disclosed, but Astellas maintains its belief that the clinical benefits of avacincaptad pegol in slowing GA lesion growth outweigh the potential risks. The drug was initially scheduled for review at the CHMP's October meeting but was subsequently removed from the agenda.
Clinical Evidence and Efficacy
Avacincaptad pegol's efficacy is supported by data from the GATHER 1 and GATHER 2 clinical trials. These studies demonstrated that intravitreal injections of avacincaptad pegol slowed disease progression by up to 35% over 12 months compared to sham control. This reduction in GA lesion growth is a key factor that Astellas believes differentiates avacincaptad pegol from other therapies, such as Syfovre (pegcetacoplan).
Market Context and Unmet Needs
Geographic atrophy affects an estimated 1.5 million people in the United States, with a significant portion remaining undiagnosed. The condition leads to severe and irreversible visual impairment and blindness. Currently, treatment options outside the US are limited, highlighting the unmet medical need that avacincaptad pegol aimed to address. The market for GA treatments is potentially substantial, with analysts previously predicting blockbuster sales for both Izervay and Syfovre. However, Syfovre has faced challenges related to reported side effects, impacting sales forecasts.
Astellas' Future Plans
Despite the EMA's decision, Astellas has affirmed its commitment to bringing avacincaptad pegol to market in Europe. The company is currently reviewing the potential financial impact of the withdrawal on its fiscal year ending March 31, 2025. Further details regarding Astellas' strategy for refiling the application in Europe have not yet been disclosed.
