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Also known as venglustat (GZ402671)
venglustat · 5 trials · 5 indications
| Arm | Type | Description |
|---|---|---|
| Venglustat | EXPERIMENTAL | Participants will receive venglustat once daily, orally |
| Standard of Care Therapy | ACTIVE_COMPARATOR | Participants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information) |
| Cerezyme | ACTIVE_COMPARATOR | Cerezyme |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo once daily for 12 months |
| Open label (OL) venglustat | EXPERIMENTAL | Administered once a day orally for up to approximately 10 years. Participants will continue their usual dose of Cerezyme during Part 1, Part 2 and Part 3. There is no administration of Cerezyme in Part 4 unless administrated as rescue treatment. |
| Participants with mild hepatic impairment | EXPERIMENTAL | Venglustat single dose on Day 1 |
| Participants with normal hepatic function | EXPERIMENTAL | Venglustat single dose on Day 1 |
| Participants with moderate hepatic impairment | EXPERIMENTAL | Venglustat single dose on Day 1 |
| Participants with severe hepatic impairment | EXPERIMENTAL | Venglustat single dose on Day 1 |
| Name | Type | Description |
|---|---|---|
| Venglustat (GZ402671) | DRUG | Tablet; Oral |
| Agalsidase alfa | DRUG | Concentrate for solution for infusion; IV infusion |
| Agalsidase beta (GZ419828) | DRUG | Powder for concentrate for solution for infusion; IV infusion |
| Migalastat | DRUG | Hard capsules; Oral |
| Venglustat | DRUG | tablet; oral |
| imiglucerase | DRUG | sterile lyophilized product; intravenous |
| Placebo | DRUG | Pharmaceutical form: Tablet Route of administration: Oral |
Inclusion Criteria: * Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease. * Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated. * Left vent...
Venglustat is an investigational small molecule being developed for rare lysosomal storage disorders, specifically Fabry disease, Gaucher disease type 1, and Gaucher disease type 3. It is also being studied in adults with hepatic impairment to assess how the drug's pharmacokinetics are affected. Venglustat has received Fast Track and Orphan Drug designations from the FDA.
Venglustat targets UDP-glucose ceramide glucosyltransferase, also known as UGCG. It acts as an inhibitor of this enzyme, which is involved in the synthesis of glucosylceramide, a key glycosphingolipid that accumulates in diseases such as Fabry and Gaucher disease. By inhibiting UGCG, venglustat aims to reduce the production of these harmful substrates.
Venglustat is being developed by Sanofi, which trades under the ticker symbol SNY. Sanofi is conducting clinical trials of venglustat in multiple countries for indications including Fabry disease and Gaucher disease type 3.
Venglustat is in Phase 3 clinical development. It is currently being evaluated in active Phase 3 trials for Fabry disease and Gaucher disease type 3, and a Phase 1 trial in hepatic impairment has been completed. Venglustat is investigational and has not been approved by the FDA.
Venglustat is being studied in several clinical trials. Key active Phase 3 trials include NCT05280548, which evaluates the effect of venglustat on left ventricular mass index in adults with Fabry disease, NCT05206773, which assesses its effect on neuropathic and abdominal pain in Fabry disease, and NCT05222906, which tests its efficacy and safety in Gaucher disease type 3. A completed Phase 1 trial, NCT05718258, investigated the impact of hepatic impairment on venglustat pharmacokinetics.
Yes, venglustat is also known by its development code GZ402671. Venglustat (GZ402671) is the same investigational drug developed by Sanofi. References to either name in scientific literature or clinical trial records refer to the same compound.