Sanofi Introduces Breakthrough Venglustat Therapy for Treating Type 3 Gaucher Disease
Sanofi has introduced venglustat for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), a rare inherited lysosomal storage disorder.
Venglustat is an investigational oral glucosylceramide synthase inhibitor (GCSi) designed to reduce the abnormal accumulation of glycosphingolipids (GSLs), which contribute to disease progression. The therapy is able to cross the blood-brain barrier, allowing it to target the neurological complications associated with GD3.
The designation is supported by results from the Phase 3 LEAP2MONO study, in which patients treated with venglustat demonstrated statistically significant improvements in neurological symptoms compared with those receiving enzyme replacement therapy (ERT) with imiglucerase. Improvements were measured using assessments of coordination, balance and cognitive function. The treatment was generally well tolerated, with no new safety concerns identified during the study.
Type 3 Gaucher disease is a rare genetic disorder caused by a deficiency of the enzyme glucocerebrosidase, leading to the accumulation of fatty substances known as glycosphingolipids in various organs and tissues. In addition to systemic symptoms such as enlargement of the liver and spleen, anaemia, low platelet counts and bone complications, patients with GD3 experience neurological manifestations that can progressively affect movement, coordination and cognitive function. While enzyme replacement therapies are available to manage systemic symptoms, there are currently no approved treatments specifically targeting the neurological aspects of the disease.
Venglustat has previously received Fast Track designation from the FDA and orphan drug designation in the United States, European Union and Japan for the treatment of GD3. Global regulatory submissions for the therapy are planned during 2026.
Venglustat is being developed as a brain-penetrant therapy aimed at slowing disease progression by reducing the build-up of glycosphingolipids, which are linked to cellular dysfunction and disease progression in several rare disorders.
