Astellas’ IZERVAY Gets FDA Approval for Expanded Label to Treat Geographic Atrophy

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Astellas Pharma announced on Feb. 13, 2025 that FDA has updated the labeling for avacincaptad pegol intravitreal solution (brand name IZERVAY) by approving the drug to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD) without a limitation on duration of dosing, which offers greater flexibility for managing GA by allowing longer-term treatment (1).

This approval follows a resubmission on Dec. 26, 2024 of a supplemental new drug application (sNDA) for avacincaptad pegol intravitreal solution. The sNDA was resubmitted within days of a meeting with FDA to clarify feedback that the agency provided in a complete response letter issued to Astellas in November 2024 (2).

“We are pleased with [FDA’s] decision to extend the use of IZERVAY for longer-term administration—further solidifying IZERVAY’s status as a trusted choice for thousands of GA patients since its launch in 2023. To date, IZERVAY remains the only FDA-approved treatment to demonstrate a statistically significant slowing of GA across two pivotal studies,” said Marci English, senior vice-president, Biopharma and Ophthalmology Development, Astellas Pharma, in a company press release (1).

AMD is the major cause of moderate and severe blindness in aging adults (3). It affects both eyes in the majority of patients. As AMD progresses, loss of retinal cells and the underlying blood vessels in the macula leads to significant thinning and/or atrophy of retinal tissue. GA, which is associated with AMD, leads to further irreversible loss of vision AMD patients. GA is defined by the presence of “sharply demarcated atrophic lesions of the outer retina, resulting from loss of photoreceptors, retinal pigment epithelium, and underlying choriocapillaris” (4).

The approval for the expanded label was based on positive results from a Phase III clinical trial (GATHER2) that evaluated the efficacy and safety of avacincaptad pegol intravitreal solution through year two of treatment. In the Phase III trial, avacincaptad pegol intravitreal solution demonstrated a continued reduction to the rate that GA lesions grew in patients with GA secondary to AMD through two years versus a sham procedure. The benefit with avacincaptad pegol intravitreal solution treatment versus sham was observed as early as six months, and the benefit continued to increase over time through two years of treatment. The benefit more than doubled over two years compared to one year of treatment with the drug.

The drug met its primary objective at the 12-month mark in the trial, for which patients were randomized to receive either avacincaptad pegol intravitreal solution or sham procedure monthly. In the second year of the study, patients treated with the drug in the first year were re-randomized to receive either avacincaptad pegol intravitreal solution dosed monthly or every other month. Meanwhile, the patients who received sham in the first year continued to receive sham in the second year of the study. Avacincaptad pegol intravitreal solution continues to be evaluated in an open-label extension study.

The drug has had month-over-month growth in the United States since April 2024 when it received a permanent government reimbursement code (J-code) (5). More than 210,000 vials were distributed from April 2024 through the end of December 2024 (1).

“This is a welcome update for retina specialists, providing continued management options for treatment of this chronic, progressive disease that can lead to irreversible vision loss,” said Veeral S. Sheth, MD, partner and director of Clinical Trials University Retina, in the press release.

Astellas first received FDA approval for avacincaptad pegol intravitreal solution (under the brand name IZERVAY) to treat GA secondary to AMD in August 2023 (6).

References

1. Astellas Pharma. US FDA Approves Expanded Label for Astellas’ IZERVAY (avacincaptad pegol intravitreal solution) for Geographic Atrophy. Press Release. Feb. 13, 2025.
2. Astellas Pharma. Astellas Provides Update on IZERVAY (avacincaptad pegol intravitreal solution) Supplemental New Drug Application. Press Release. Nov. 19, 2024.
3. Fleckenstein,M.; Schmitz-Valckenberg, S.; Chakravarthy, U. Age-Related Macular Degeneration: A Review. JAMA 2024, 331 (2),147–157. DOI:10.1001/jama.2023.26074
4. Fleckenstein, M.; Mitchell, P.; Freund, K. B.; et al. The Progression of Geographic Atrophy Secondary to Age-Related Macular Degeneration. Ophthalmology 2018, 125 (3), 369–390. DOI: 10.1016/j.ophtha.2017.08.038
5. Centers for Medicare & Medicaid Services. Healthcare Common Procedure Coding System (HCPCS). cms.gov (accessed Feb. 19, 2025).
6. Astellas Pharma. Iveric Bio Receives US FDA Approval for IZERVAY (avacincaptad pegol intravitreal solution), a New Treatment for Geographic Atrophy. Press Release. Aug. 5, 2023.