Introduction to Astellas’ Pioneering Regulatory Submission
According to the news (News | Astellas Pharma Inc.) released by Astellas Pharma on Feb/05/2025, they have submitted a manufacturing and marketing approval application in Japan for avacincaptad pegol (ACP), branded as IZERVAY, targeting geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Notably, this application is under Japan’s conditional approval system and is based solely on overseas clinical trial data, marking a potential first in the Japanese pharmaceutical landscape.
Understanding Geographic Atrophy and the Need for Treatment
GA is an advanced form of AMD leading to irreversible vision loss. Currently, there are no approved treatments for GA in Japan, underscoring a significant unmet medical need. Globally, over 5 million individuals are affected by GA, with a substantial proportion at risk of severe visual impairment if untreated.
Clinical Evidence Supporting the Application
The submission is made based on data of two pivotal overseas clinical trials, GATHER1 and GATHER2, which evaluated the safety and efficacy of monthly 2 mg intravitreal injections of ACP in GA patients. Results demonstrated that ACP significantly slowed GA progression with a favorable safety profile, with effects sustained over a two-year period.
Implications of the Conditional Approval System
Japan’s conditional approval system expedites access to therapies addressing serious conditions with high unmet needs, even when Japanese clinical data is lacking. Astellas’ reliance on international trial data for ACP’s approval could set a precedent, potentially streamlining the introduction of innovative treatments into the Japanese market.
Conclusion
Astellas’s application for ACP under Japan’s conditional approval framework, without Japanese patient data, represents a significant regulatory milestone. If approved, IZERVAY would be the first treatment available in Japan for GA secondary to AMD, offering hope to patients facing this debilitating condition.