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) caused by age-related macular degeneration (AMD). The fast track status is designed to accelerate the development and review of therapies addressing serious conditions with unmet medical needs.
SAR446597 works by delivering genetic material encoding two antibody fragments that inhibit key components of the complement system—C1s in the classical pathway and factor Bb in the alternative pathway. This dual-targeted approach aims to provide sustained suppression of the complement pathway in the retina, potentially slowing disease progression while reducing the need for frequent injections.
Sanofi plans to initiate a Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of SAR446597 in patients with GA.
In addition, Sanofi is currently conducting a Phase 1/2 study of another gene therapy candidate, SAR402663, for patients with neovascular (wet) AMD, further expanding its portfolio of investigational treatments targeting retinal diseases.