Astellas Pharma US Inc. – FDA CRL

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to Astellas Pharma US, Inc. on November 15, 2024, regarding their supplemental New Drug Application (sNDA) for IZERVAY (avacincaptad pegol intravitreal solution). The sNDA, submitted on January 19, 2024, sought approval for an expanded dosing regimen beyond 12 months for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD) under section 505(b) of the Federal Food, Drug, and Cosmetic Act.The FDA determined that the proposed extended dosing interval was not supported by the submitted clinical data from the ISEE2008 (GATHER2) study. Specifically, the study showed a numerically higher GA growth rate in the every-month arm compared to the every-other-month arm, and formal statistical inference for the extended interval was not established, nor was the proposal replicated in a second adequate study.To gain approval, Astellas must submit data from new, adequate, and well-controlled studies demonstrating the efficacy of IZERVAY with an extended dosing regimen. Additionally, a comprehensive safety update is required, including new safety data, adverse event tabulations, detailed information on trial discontinuations and serious adverse events, updated exposure data, and a summary of worldwide safety experience. The company has one year to resubmit the application, which must clearly address all identified deficiencies.