The US FDA granted Breakthrough Therapy designation to Sanofi’s investigational oral venglustat for neurological manifestations of type 3 Gaucher disease, based on phase 3 LEAP2MONO study data showing statistically significant improvements (p=0.007) in ataxia and cognition versus imiglucerase. This expedites development for a condition with no approved neurological treatments.
Key takeaways
FDA Breakthrough Therapy designation for venglustat in GD3 on March 18, 2026
LEAP2MONO phase 3 study (NCT05222906): significant improvement in global test score (mSARA ataxia + RBANS cognition, p=0.007) vs ERT
Venglustat well tolerated; AEs: headache (14.3% vs 18.2%), nausea (14.3% vs 4.5%), spleen enlargement (14.3% vs 0%)
No approved treatments for GD3 neurological symptoms; Sanofi plans global filings in 2026
Prior FDA fast-track and orphan designations for GD3 in US, EU, Japan