Sanofi’s Intravitreal Gene Therapy Receives FDA Fast Track Designation for Geographic Atrophy

赛诺菲的玻璃体内基因疗法获FDA快速通道指定用于地理萎缩症

July 17, 2025

2025年7月17日

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAR446597, a one-time intravitreal gene therapy developed by Sanofi for the treatment of geographic atrophy (GA) due to age-related macular degeneration (AMD). This designation is intended to accelerate the development and review of drugs that address serious medical conditions and meet unmet clinical needs..

美国食品药品监督管理局（FDA）已授予赛诺菲开发的单次玻璃体内基因疗法SAR446597快速通道资格，该药物用于治疗因年龄相关性黄斑变性（AMD）引起的地理萎缩（GA）。该资格旨在加速针对严重医疗状况并满足未满足临床需求的药物的开发和审评进程。

Addressing an Unmet Need in GA

满足GA中未满足的需求

Geographic atrophy represents an advanced stage of AMD, for which current treatment options remain limited. The FDA’s Fast Track designation for SAR446597 highlights the potential of this novel therapy to significantly impact the clinical management of GA and improve patient outcomes. This status provides Sanofi with opportunities for more frequent engagement with the FDA, the possibility of priority review, and a faster pathway to regulatory approval..

地理萎缩代表了AMD的晚期阶段，目前的治疗选择仍然有限。 SAR446597获得FDA的快速通道资格，突显了这种新型疗法在显著影响GA临床管理及改善患者预后方面的潜力。 此资格使赛诺菲有更多机会与FDA频繁沟通，具备优先审查的可能性，并加快监管批准的进程。

Dual-Targeting Approach: A Novel Mechanism of Action

双靶向方法：一种新颖的作用机制

SAR446597 works by delivering genetic material that encodes two therapeutic antibody fragments. These fragments are designed to inhibit two key components of the complement pathway: C1s, which plays a role in the classical pathway, and factor Bb, which is part of the alternative pathway.

SAR446597通过递送编码两种治疗性抗体片段的遗传物质来发挥作用。这些片段旨在抑制补体途径的两个关键成分：C1s，它在经典途径中起作用，以及因子Bb，它是替代途径的一部分。

By targeting both pathways simultaneously, SAR446597 aims to provide more comprehensive and sustained complement suppression within the retinal microenvironment. This dual-targeting mechanism is expected to address the underlying pathophysiology of complement-mediated retinal diseases more effectively than current treatment modalities..

通过同时靶向两条通路，SAR446597旨在视网膜微环境中提供更全面和持久的补体抑制。这种双重靶向机制预计比当前的治疗方式更有效地应对补体介导的视网膜疾病的潜在病理生理机制。

Potential Clinical Benefits

潜在的临床益处

Sanofi's gene therapy approach offers several potential advantages:

赛诺菲的基因治疗 approach 提供了几个潜在的优势：

• Sustained therapeutic effect through long-term expression of the encoded proteins after a single administration

• 单次给药后，通过编码蛋白的长期表达持续产生治疗效果

• Reduced treatment burden by eliminating the need for frequent intravitreal injections

• 通过消除频繁的眼内注射需求，减轻了治疗负担

• Comprehensive complement pathway inhibition, which could result in better disease control and slower progression of GA

• 全面的补体通路抑制，可能会更好地控制疾病并减缓GA的进展

Clinical Development Pipeline

临床开发管道

Sanofi plans to initiate a phase 1/2 clinical study to evaluate the safety, tolerability, and efficacy of SAR446597 in patients with geographic atrophy. In addition, the company is actively studying another gene therapy, SAR402663, in a phase 1/2 trial for the treatment of wet AMD (ClinicalTrials.gov Identifier: NCT06660667)..

赛诺菲计划启动一项1/2期临床研究，评估SAR446597在地理萎缩患者中的安全性、耐受性和有效性。此外，该公司正在进行另一项基因疗法SAR402663的1/2期试验，用于治疗湿性AMD（ClinicalTrials.gov标识符：NCT06660667）。