FDA fast tracks one-time intravitreal gene therapy for geographic atrophy

SAR446597 is designed to inhibit C1s and factor Bb in the complement pathway. Fast track designation expedites the development and review of treatments.

The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from Sanofi.

The one-time injection is designed to target and inhibit two components of the complement pathway: C1s in the classical pathway and factor Bb in the alternative pathway.

Sanofi is planning a phase 1/2 study to investigate safety, tolerability and efficacy of SAR446597 gene therapy.

“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,” Sanofi said in the release. “The therapy aims to address the underlying pathophysiology of complement-mediated retinal diseases through long-term expression of therapeutic proteins following a single intervention.”

Fast track designation is granted to expedite the development and review of medicines that treat serious conditions and address unmet medical needs.

Sanofi aims to evaluate the safety, tolerability and efficacy of SAR446597 in an upcoming phase 1/2 study.