Astellas Pharma Inc. announced it received notification from the U.S. Food and Drug Administration (FDA) of acceptance of the company's supplemental New Drug Application (sNDA) to include positive 2-year data in the U.S. Prescribing Information for IZERVAYTM (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The sNDA is based on results from the GATHER2 Phase 3 clinical trial, which evaluated the efficacy and safety of monthly (EM) and every other month (EOM) dosing through year 2. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of November 19, 2024. The acceptance follows the U.S. Centers for Medicare and Medicaid Services issuing a permanent J-code for IZERVAY, effective April 1, which is expected to accelerate patient access in the U.S. The GATHER2 data demonstrated IZERVAY continued to reduce the rate of GA lesion growth in patients with GA secondary to AMD through 2 years versus sham.

The treatment benefit with IZERVAY vs. sham was observed as early as 6 months, continued to increase over time through 2 years, and more than doubled over 2 years compared to year 1. IZERVAY was well tolerated over 2 years in GATHER2, with one case of non-serious intraocular inflammation (IOI) and culture-positive endophthalmitis each, and zero cases of ischemic neuropathy or serious intraocular inflammation, including retinal vasculitis. Over 2 years, the incidence of choroidal neovascularization (CNV) was slightly increased between IZERVAY (11.6%) versus sham (9%).

IZERVAY was approved by the U.S. Food and Drug Administration on August 4, 2023, for the treatment of GA secondary to AMD and is currently under review by the European Medicines Agency. Astellas has already reflected the impact from this result in its financial forecast for the fiscal year ending March 31, 2024. GATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD.

ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly. In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP dosed monthly (EM, n=96) or every other month (EOM, n=93); patients who received sham in year 1 continued to receive sham in year 2 (n=203). IZERVAY is continuing to be evaluated in an open-label extension study.