Sanofi: FDA Grants Priority Review for Venglustat for Gaucher's Disease
On Thursday, the FDA granted a priority review for the approval of venglustat to treat type 3 Gaucher's disease in the United States. If approved, this oral treatment would be the first in the country specifically targeting the neurological manifestations of this rare disease.
A Treatment Targeting Neurological Symptoms
Venglustat is a novel oral glucosylceramide synthase inhibitor (GCSi) designed to cross the blood-brain barrier. Type 3 Gaucher's disease is characterized by an abnormal accumulation of sugar and fat molecules in the spleen, liver, bone marrow, and lungs, as well as in the central nervous system where it causes neuronal inflammation that can lead to cognitive deficits and coordination and balance disorders.
Currently, no targeted treatment exists for the neurological symptoms of this rare disease. Venglustat has the potential to treat these manifestations by crossing the brain barrier.
Clinical Data and Previous Approvals
The approval application is supported by positive data from the phase 3 LEAP2MONO study, which evaluated the efficacy and safety of venglustat in adults and pediatric patients with neurological manifestations of type 3 Gaucher's disease, who had previously achieved stabilization of systemic manifestations through enzyme replacement therapy.
Venglustat has already received the FDA's breakthrough therapy designation and a fast track designation, as well as orphan drug designation in the United States, the European Union, and Japan. The target date for the FDA's decision is set for November 25, 2026. Sanofi plans to continue its regulatory filings in other regions of the world throughout the year 2026.
