Sanofi’s Venglustat accepted for priority review to treat type 3 Gaucher disease in the US

The US Food and Drug Administration (FDA) has granted priority review to Sanofi’s new drug application for venglustat, a novel investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder.

If approved, venglustat would become the first treatment available in the US to address the progressive neurological manifestations of GD3 and would expand Sanofi’s portfolio of treatment options for patients living with lysosomal storage diseases. The FDA target action date is set for November 25, 2026. Gaucher disease is characterised by the abnormal accumulation of sugar and fat molecules known as glycosphingolipids (GSL) in the spleen, liver, bone marrow, and lungs. In patients with GD3, these molecules also build up in the central nervous system (CNS), where they drive neuroinflammation and can lead to neurological manifestations such as cognitive deficits and difficulty with coordination and balance (ataxia), on top of the disease’s systemic effects. At present, there are no approved targeted therapies that specifically address the neurological symptoms of GD3. By crossing the blood brain barrier, venglustat has the potential to treat these neurological manifestations.

The new drug application is supported by positive data from the LEAP2MONO Phase 3 study (clinical study identifier NCT05222906), which evaluated the efficacy and safety of venglustat in adult and paediatric patients with neurological manifestations of GD3 who had previously achieved stabilisation of systemic manifestations on enzyme replacement therapy (ERT). In results shared at the WORLDSymposium earlier this year, venglustat met both primary endpoints and three of the four key secondary endpoints. In the study, venglustat was well tolerated overall, with no new safety signals identified compared with previous studies. The most commonly reported adverse events were headache (14.3% in the venglustat arm versus 18.2% in the ERT arm), nausea (14.3% versus 4.5%), spleen enlargement (14.3% versus 0%), and diarrhoea (14.3% versus 0%).

Venglustat has previously received breakthrough therapy designation and fast track designation from the FDA for its potential in GD3, as well as orphan designation for GD3 in the US, the EU, and Japan. The medicine is also currently under regulatory review for GD3 in the EU, and Sanofi intends to pursue additional global regulatory filings for venglustat in GD3 across the remainder of 2026.

The safety and efficacy of venglustat for GD3 have not yet been evaluated by any regulatory authority. Priority review is granted to regulatory applications seeking approval for therapies that have the potential to deliver significant improvements in the treatment, diagnosis, or prevention of serious conditions.

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FDA Approval, Sanofi, Sanofi’s venglustat, type 3 Gaucher disease, Venglustat