Sanofi Secures FDA Breakthrough Therapy Status for Venglustat in Type 3 Gaucher Disease
The US Food and Drug Administration (FDA) has awarded Breakthrough Therapy designation to venglustat, an investigational oral therapy, for the treatment of neurological symptoms in type 3 Gaucher disease (GD3).
GD3 is a rare inherited disorder that affects the brain and other organs, including the liver, spleen, and bone marrow.
The designation follows results from the phase 3 LEAP2MONO study, which showed that patients taking venglustat had significant improvements in neurological function, including movement coordination and cognition, compared with standard enzyme replacement therapy (ERT).
Venglustat was generally well tolerated, with headaches, nausea, spleen enlargement, and diarrhoea being the most common side effects.
While ERT addresses systemic symptoms of GD3, there are currently no approved treatments for the neurological effects.
Venglustat is designed to cross the blood-brain barrier and target the underlying cause of neurological damage.
Venglustat has already received fast-track and orphan drug designations in the US, EU, and Japan. Sanofi plans to pursue global regulatory submissions for GD3 during 2026.
The LEAP2MONO study is ongoing, and additional results from its open-label phase will be shared when available.
