Sanofi's SAR446597 receives US fast track status for age-related macular degeneration

Sanofi's SAR446597, an innovative gene therapy developed for the treatment of geographic atrophy (GA) associated with age-related macular degeneration (AMD), has received the fast track designation from the US Food and Drug Administration (FDA). This designation aims to accelerate the development and review processes of therapies addressing critical medical needs and serious health conditions.

GA is an advanced stage of dry AMD, characterized by irreversible retinal cell degeneration leading to significant vision loss. Affecting approximately one million people in the United States alone, GA has a substantial impact on the quality of life, impairing fundamental abilities like reading and driving. Globally, AMD affects around 200 million individuals, with GA impacting over 5 million people worldwide.

The novel therapy, SAR446597, employs a unique intravitreal approach, introducing genetic material that encodes therapeutic antibodies. These antibodies target two essential components of the complement cascade: C1s in the classical pathway and factor Bb in the alternative pathway. By inhibiting these pathways, SAR446597 aims to provide sustained suppression of complement activity in the retinal environment. This dual-targeting strategy could offer significant clinical benefits, potentially reducing the frequency of treatments and addressing the root causes of complement-mediated retinal disorders.

Sanofi plans to commence a phase 1/2 clinical trial to assess the safety, tolerability, and effectiveness of SAR446597. This trial represents an essential step in determining the therapy's potential benefits for patients dealing with GA due to AMD.

In parallel, Sanofi is advancing its research on another gene therapy, SAR402663, currently in a phase 1/2 study for treating neovascular wet AMD. This research highlights Sanofi's commitment to exploring pioneering treatments for ophthalmological conditions with unmet medical needs, particularly those intersecting with immune system disorders.

Sanofi's broader mission in neurology encompasses addressing severe neuroinflammatory and neurodegenerative diseases. The company is dedicated to advancing treatments for conditions such as multiple sclerosis, chronic inflammatory demyelinating polyneuropathy, Alzheimer's, Parkinson's, and AMD. The integration of emerging scientific innovations and strategic investments in the field of ophthalmology positions Sanofi to drive a new phase of growth within the sector.

As a research and development-focused biopharmaceutical organization, Sanofi leverages cutting-edge artificial intelligence technologies and profound insights into the immune system to develop vaccines and medications. These innovations aim to improve and protect the lives of millions globally, reflecting the company's commitment to pursuing scientific breakthroughs that address pressing health, environmental, and societal challenges.

Sanofi remains steadfast in its purpose-driven approach, aiming to deliver substantial positive impacts for communities worldwide while addressing crucial healthcare needs. By focusing on groundbreaking scientific advancements, Sanofi continues to make strides in developing therapies that could significantly alter the landscape of treatment for serious and chronic health conditions.