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Venglustat GZ402671
Venglustat is a selective and reversible inhibitor of glucosylceramidase.
Venglustat is a selective and reversible inhibitor of glucosylceramidase. Used for Treatment of adult patients with type 1 diabetes mellitus with a history of severe hypoglycemia requiring medical intervention.
At a glance
| Generic name | Venglustat GZ402671 |
|---|---|
| Sponsor | Sanofi |
| Drug class | GLP-1 receptor agonist |
| Target | GLP-1R |
| Modality | Small molecule |
| Therapeutic area | Diabetes |
| Phase | Phase 3 |
Mechanism of action
By inhibiting glucosylceramidase, venglustat reduces the accumulation of glucosylceramide in cells, which is associated with Fabry disease. This mechanism of action is thought to provide therapeutic benefits for patients with Fabry disease.
Approved indications
- Treatment of adult patients with type 1 diabetes mellitus with a history of severe hypoglycemia requiring medical intervention
Common side effects
- Nausea
- Diarrhea
- Vomiting
- Abdominal pain
- Headache
- Injection-site reaction
Key clinical trials
- A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (PHASE3)
- A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease (PHASE3)
- A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (PHASE3)
- Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat (PHASE3)
- A Study in Adults to Investigate the Impact of Mild, Moderate, and Severe Hepatic Impairment on Pharmacokinetics of Venglustat Compared to Participants With Normal Hepatic Function (PHASE1)
- Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension (PHASE2)
- A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients (PHASE2, PHASE3)
- Effect of Venglustat in Patients With Renal Impairment (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Venglustat GZ402671 CI brief — competitive landscape report
- Venglustat GZ402671 updates RSS · CI watch RSS
- Sanofi portfolio CI