FDA Grants Fast Track Status to Sanofi’s Gene Therapy Candidate for Wet AMD

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Sanofi’s investigational gene therapy, SAR402663, for the treatment of neovascular (wet) age-related macular degeneration (AMD). The therapy is currently being evaluated in a Phase 1/2 clinical trial for its potential to reduce the treatment burden and provide long-term disease control.

The FDA’s Fast Track Designation is intended to accelerate the development and review of new therapies targeting serious conditions with unmet medical needs. The designation enables closer communication with the FDA, with the goal of bringing important treatments to patients more rapidly.

Overview of SAR402663: Investigational Intravitreal Gene Therapy

SAR402663 is a single-dose gene therapy administered via intravitreal injection. The therapy is designed to deliver genetic instructions encoding soluble FLT01, a protein engineered to inhibit vascular endothelial growth factor (VEGF), a primary driver of abnormal blood vessel growth and leakage in wet AMD.

By targeting the underlying disease pathology, SAR402663 aims to prevent abnormal choroidal neovascularization, reduce vascular leakage and retinal damage, and lower the treatment burden by potentially eliminating the need for frequent intravitreal anti-VEGF injections.

Ongoing Clinical Evaluation

Sanofi is currently evaluating SAR402663 in a Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT06660667) to assess its safety, tolerability, and preliminary efficacy in patients with wet AMD.

If proven effective, SAR402663 could offer long-term VEGF suppression from a single administration, significantly reducing or even eliminating the need for monthly intravitreal anti-VEGF injections, a major limitation of current therapies.