Drugs / Venglustat
Venglustat
also known as
GZ402671 · GZ/SAR402671 · Genz-682452 · Genz-682452-AA · Genz-682452-AU · GZ/SAR-402671A · Ibiglustat · Ibiglustat l-malate · +4 more
Small molecule targets UGCG via inhibition
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Gaucher disease type III | 2026-05-28 | “The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for venglustat” sanofi.com ↗ |
| Filed | EU (EMA) | — | Gaucher disease type III | — | “Venglustat is also currently under regulatory review for GD3 in the EU.” sanofi.com ↗ |
Trials 18
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 17 trials | ||||||
| Phase 1 | NCT05718258 | Jan 2023 → Jan 2024 | liver disorder | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT06418620 | Jun → Aug 2020 | — | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT05238714 | May → Jun 2020 | sphingolipidosis | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT03687554 | Oct 2018 → Feb 2019 | autosomal dominant polycystic kidney disease | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT06421714 | Aug → Oct 2018 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT01710826 | Oct 2012 → Feb 2013 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 1 | NCT01674036 | Aug 2012 → Jan 2013 | — | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 24 trials · 2 missed | ||||||
| Phase 2 | NCT02843035 | Jan 2017 → Oct 2026 expected | Gaucher disease type I, Gaucher disease type III | Genzyme, a Sanofi Company | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02906020 | Dec 2016 → Dec 2020 | Parkinson disease | Genzyme, a Sanofi Company | Terminated | Missed primary |
| Phase 2 | NCT02489344 | Jul 2015 → Nov 2018 | Fabry disease | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 2 | NCT02228460 | Nov 2014 → Sep 2016 | Fabry disease | Genzyme, a Sanofi Company | Completed | Missed primary |
| Phase 2/31 trial · 1 missed | ||||||
| Phase 2/3 | NCT03523728 | Oct 2018 → Aug 2021 | autosomal dominant polycystic kidney disease | Genzyme, a Sanofi Company | Terminated | Missed primary |
| Phase 35 trials · 1 met primary | ||||||
| Phase 3 | NCT05280548 | May 2022 → May 2026 overdue | Fabry disease | Sanofi | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05222906 | Apr 2022 → Oct 2025 overdue | Gaucher disease type III | Sanofi | Active not recruiting | Met primary |
| Phase 3 | NCT05206773 | Mar 2022 → Dec 2025 overdue | Fabry disease | Sanofi | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04705051 | Feb → Jul 2021 | cystic kidney disease | Sanofi | Terminated | No outcome recorded |
| Phase 3 | NCT04221451 | Jun 2020 → Dec 2024 | Sandhoff disease, Tay-Sachs disease | Genzyme, a Sanofi Company | Terminated | Mixed |
| Phase not stated1 trial | ||||||
| — | NCT07675031 | — | Gaucher disease type III | Sanofi | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2026-05-28 | Sanofi | Regulatory Press Release: Sanofi’s venglustat accepted for priority review in the US to treat type 3 Gaucher disease sanofi.com ↗ |
| 2026-03-18 | Sanofi | Regulatory Press Release: Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease sanofi.com ↗
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. |
| 2026-02-02 | Sanofi | Results Press Release: Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease sanofi.com ↗
Venglustat demonstrated superiority versus enzyme replacement therapy in addressing neurological symptoms in GD3, for which there are no approved treatments |
| 2021-06-01 | Sanofi | Results Sanofi provides update on venglustat clinical program sanofi.com ↗
The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo. |
| 2021-02-02 | Sanofi | Results Media Update: Data presented at WORLDSymposiumTM reinforces robust rare disease pipeline and highlights additional clinical data for investigational avalglucosidase alfa in Pompe disease sanofi.com ↗
Venglustat Combined with imiglucerase positively affects neurological features and brain connectivity in adult with Gaucher disease type 3 (Encore) |
All press releases naming this drug 5 releases
Evidence & citations 17 cited values
Every value below carries the sentence it was read from. 21 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Venglustat | “Venglustat, an orally administered glucosylceramide synthase inhibitor:” PMID 36481125 ↗ Nov 202214“The program provides access to venglustat, an investigational therapy.” NCT07675031 ↗ |
| Known as | Genz-682452 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01710826 ↗ |
| Known as | Genz-682452-AA | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | Genz-682452-AU | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | GZ/SAR-402671A | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | GZ/SAR402671 | “A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation” NCT02906020 ↗4“Early Versus Delayed Treatment With Venglustat (GZ/SAR402671) in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD).” NCT04705051 ↗ “Venglustat GZ/SAR402671” NCT03687554 ↗ |
| Known as | GZ402671 | “Venglustat (GZ402671)” NCT05280548 ↗5“Venglustat (GZ402671)” NCT05206773 ↗ “Venglustat (GZ402671)” NCT05718258 ↗ “venglustat (GZ402671)” NCT02843035 ↗ “venglustat GZ402671” NCT04221451 ↗ “Venglustat GZ402671” NCT04705051 ↗ |
| Known as | Ibiglustat | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | Ibiglustat l-malate | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | SAR-402671 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | SAR-402671A | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | Venglustat l-malate | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Known as | VENGLUSTAT MALATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297611 ↗ |
| Action | Inhibit | “Venglustat inhibits the enzymatic conversion of ceramide to glucosylceramide” PMID 36481125 ↗ Nov 20221“Ceramide glucosyltransferase inhibitor” CHEMBL4297611 ↗ |
| Modality | Small molecule | “Venglustat is a brain-penetrant, small molecule inhibitor of glucosylceramide synthase” PMID 37050861 ↗ Apr 2023 |
| Route | Oral | “Route of administration: Oral” NCT02228460 ↗ |
| Target | UGCG | “Ceramide glucosyltransferase inhibitor” CHEMBL4297611 ↗ |