Posted by Michael Wonder on 18 Mar 2026
Sanofi’s venglustat earns breakthrough therapy designation in the US for type 3 Gaucher disease
18 March 2026 - The US FDA has granted breakthrough therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor, for the treatment of neurological manifestations of type 3 Gaucher disease, a rare lysosomal storage disorder.
The designation is based on data from the LEAP2MONO Phase 3 study in which patients receiving venglustat demonstrated statistically significant improvements in neurological symptoms measured by a global test score that included assessments for ataxia (mSARA) and cognition (RBANS) compared with patients receiving the enzyme replacement therapy, imiglucerase (p=0.007).
