Sanofi's SAR446597 gets fast-track designation for age-related macular degeneration treatment.

Sanofi's SAR446597, a one-time intravitreal gene therapy, has been granted fast track designation by the US FDA for the treatment of geographic atrophy due to age-related macular degeneration. This designation aims to expedite the review of medicines for serious conditions and fill unmet medical needs. SAR446597 targets and inhibits two key complement cascade pathways, with the goal of preserving vision and preventing permanent vision loss.

Sanofi's SAR446597, a one-time intravitreal gene therapy, has been granted fast track designation by the US Food and Drug Administration (FDA) for the treatment of geographic atrophy (GA) due to age-related macular degeneration (AMD). This designation, aimed at expediting the review of medicines for serious conditions and filling unmet medical needs, is a significant milestone in the development of the therapy.

SAR446597 delivers genetic material encoding two therapeutic antibody fragments that target and inhibit two critical components of the complement pathway: C1s in the classical pathway and factor Bb in the alternative pathway. This dual-targeting approach potentially offers clinical advantages by providing sustained complement suppression within the retinal microenvironment while significantly reducing treatment burden through elimination of frequent intravitreal injections. The therapy aims to address the underlying pathophysiology of complement-mediated retinal diseases through long-term expression of therapeutic proteins following a single intervention.

Geographic atrophy is an advanced form of dry AMD, characterized by enlarging irreversible atrophic lesions due to degeneration of retinal cells leading to permanent vision loss in many patients. GA affects approximately 1 million people in the US, more than 2.5 million in Europe, and over 5 million people worldwide, having a profound impact on quality of life. The fast track designation for SAR446597 is expected to accelerate its development and approval process, potentially offering a breakthrough treatment for patients suffering from this condition.

Sanofi plans to start a phase 1/2 study to evaluate the safety, tolerability, and efficacy of SAR446597. This study will provide critical data to support further clinical development and regulatory submissions. Additionally, Sanofi is currently evaluating SAR402663, another one-time intravitreal gene therapy, in a phase 1/2 study for the treatment of neovascular wet age-related macular degeneration.

Sanofi's commitment to improving the lives of people with serious neuroinflammatory and neurodegenerative diseases is evident through its innovative pipeline in ophthalmology. The company is exploring therapies that address unmet needs in retinal diseases, particularly those connected with immune system conditions.

References:
[1] https://www.globenewswire.com/news-release/2025/07/16/3116124/0/en/Press-Release-Sanofi-s-SAR446597-earns-fast-track-designation-in-the-US-for-geographic-atrophy-due-to-age-related-macular-degeneration.html
[2] https://www.prnewswire.com/news-releases/artificial-intelligence-revolutionizing-early-detection-of-diabetic-retinopathy-creating-a-multi-billion-dollar-revenue-opportunity-302505344.html