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

The FDA has granted fast track designation to SAR446597, a one-time intravitreal gene therapy for the treatment of geographic atrophy (GA) due to age-related macular degeneration (AMD). The fast track designation process aims to facilitate the development and expedite the review of medicines to treat serious conditions and fill unmet medical need. 

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. According to Sanofi, this dual-targeting approach potentially offers clinical advantages by providing sustained complement suppression within the retinal microenvironment while 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.

Sanofi plans to start a phase 1/2 study to evaluate the safety, tolerability, and efficacy of SAR446597. The company is also currently evaluating SAR402663, a one-time intravitreal gene therapy, in a phase 1/2 study (clinical study identifier: NCT06660667), for the treatment of patients with wet age-related macular degeneration (AMD).