Health
Press Release: Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease
Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease Paris, March 18, 2026. The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. The designation is based on data from the LEAP2MONO phase 3 study (clinical study identi
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