Sanofi Wins US Breakthrough Therapy Tag for Venglustat in Type 3 Gaucher Disease
The US Food and Drug Administration (FDA) has awarded Breakthrough Therapy designation to venglustat, an investigational oral glucosylceramide synthase inhibitor (GCSi), for treating neurological symptoms associated with type 3 Gaucher disease (GD3), a rare lysosomal storage disorder.
This designation is supported by findings from the Phase 3 LEAP2MONO trial (NCT05222906), where patients treated with venglustat showed statistically significant improvements in neurological outcomes. These were measured using a composite global test score incorporating ataxia (mSARA) and cognitive fun...
