Tag Archives: Gaucher disease type 3

Sanofi Receives FDA Breakthrough Therapy Designation for Venglustat Targeting Neurological Symptoms of Type 3 Gaucher Disease

Posted on 18, March 2026 by EuropaWire PR Editors

(IN BRIEF) Sanofi has received Breakthrough Therapy designation from the US FDA for venglustat, an investigational oral drug targeting neurological symptoms of type 3 Gaucher disease, a rare genetic disorder with no approved treatments for its neurological manifestations. The designation … Read the full press release →

Posted in European Union, France, Healthcare, Marketing, Pharma & Biotech

Tagged biotechnology, clinical trials, enzyme replacement therapy, Fast Track Designation, FDA, Gaucher disease type 3, GD3, imiglucerase, Karin Knobe, LEAP2MONO, lysosomal storage disorder, orphan drug, rare disease, Sanofi, Sanofi Strengthens Pipeline with Venglustat Following FDA Breakthrough Designation for Rare Neurological Disorder, US Food and Drug Administration, venglustat