
Sanofi has received the US Food and Drug Administration (FDA) fast track designation for SAR446597, a one-time intravitreal gene therapy for treating geographic atrophy (GA) due to age-related macular degeneration (AMD).
The fast-track designation is granted for treatments addressing serious conditions with unmet medical needs, aiming to deliver new drugs to patients more swiftly.
SAR446597 is designed to address the underlying pathophysiology of complement-mediated retinal diseases, providing long-term expression of therapeutic proteins.
The drug works by delivering genetic material that encodes two therapeutic antibody fragments.
The fragments target and inhibit C1s in the classical pathway and factor Bb in the alternative pathway of the complement cascade.
The dual-targeting strategy offers clinical benefits by maintaining complement suppression in the retinal microenvironment and reducing the frequency of intravitreal injections.
Sanofi plans to initiate a Phase 1/2 study to assess the safety, tolerability, and efficacy of SAR446597.
In addition, Sanofi is evaluating SAR402663, another one-time intravitreal gene therapy, in a Phase 1/2 study for patients with neovascular wet age-related macular degeneration.
AMD is a progressive retinal degeneration condition, with geographic atrophy being an advanced form of dry AMD leading to permanent vision loss.
In May, Sanofi agreed to acquire Vigil Neuroscience, a clinical-stage biotechnology company dedicated to developing therapies for neurodegenerative diseases.
One month later, the French pharmaceutical company agreed to acquire US-based Blueprint Medicines for about $9.5bn to enhance its portfolio in rare immunological diseases.