Sanofi AAV Gene Therapy Receives FDA Fast Track Designation

Paris, September 11, 2025 – The US Food and Drug Administration (FDA) has granted fast track designation to SAR402663, an investigational one-time intravitreal gene therapy for the treatment of neovascular 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. The FDA created this process to help deliver important new drugs to patients earlier and it covers a broad range of serious illnesses.

SAR402663, which utilizes an adeno-associated virus (AAV) vector, delivers genetic material encoding soluble FLT01 designed to inhibit vascular endothelial growth factor (VEGF). Sanofi is currently evaluating SAR402663 in a phase 1/2 (clinical study identifier: NCT06660667), for the treatment of patients with neovascular AMD. The gene therapy aims to address the underlying disease pathology by inhibiting abnormal blood vessel growth, reducing vascular leakage and minimizing retina damage, while significantly reducing treatment burden through the elimination of frequent intravitreal injections.

AMD is an acquired progressive degeneration of the retina that affects approximately 200 million people globally. Neovascular, or “wet”, AMD is a severe form of macular degeneration. It is characterized by growth of abnormal blood vessels beneath the retina, which can lead to vision loss, potentially progressing to blindness in advanced cases. Neovascular AMD affects more than one million people in the US and six million people worldwide; it has a profound impact on quality of life, including ability to read, drive and perform other daily activities.