Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as venglustat (GZ402671), Venglustat (GZ402671)
Venglustat · 9 trials · 7 indications
Maximum plasma or blood concentration observed
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time, tlast (time corresponding to the last concentration above the limit of quantification, Clast)
Time to Cmax
Time to reach Cmax
| 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 |
| Sequence 1 | EXPERIMENTAL | Venglustat hard capsule administered in period 1 followed by tablet administered in period 2. |
| Sequence 2 | EXPERIMENTAL | Venglustat tablet administered in period 1 followed by hard capsule administered in period 2. |
| Treatment A | EXPERIMENTAL | Fasted, venglustat tablet swallowed with water |
| Treatment B | EXPERIMENTAL | Fasted, venglustat tablet chewed and swallowed without water |
| Treatment C | EXPERIMENTAL | Fed, venglustat tablet swallowed with water |
| [14C] venglustat | EXPERIMENTAL | Single dose of \[14C\] venglustat Oral Solution under fasting conditions |
| Venglustat and Itraconazole | EXPERIMENTAL | The first period will include a single dose administration of venglustat followed by a second period including a multiple dose administration of itraconazole for 12 days and a second single dose administration of venglustat on the 6th day of itraconazole administration |
| 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 |
| Itraconazole | DRUG | Pharmaceutical form:Capsule-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 diseases, including Gaucher Disease Type 1, Gaucher Disease Type 3, and Fabry Disease. It is also studied in healthy volunteers and in patients with hepatic function abnormalities or disorders of sphingolipid metabolism.
Venglustat is a small molecule that belongs to the -stat class of enzyme inhibitors. It targets enzymes involved in sphingolipid metabolism, which are implicated in the pathology of Gaucher and Fabry diseases.
Venglustat is developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker SNY. Sanofi is conducting clinical trials to evaluate the drug's safety and efficacy in multiple rare disease indications.
Venglustat is in Phase 3 clinical development for Fabry Disease and Gaucher Disease Type 3. It has received FDA designations including Breakthrough Therapy, Fast Track, Orphan Drug, and Priority Review, but it remains investigational and is not yet approved.
Venglustat is being studied in several active Phase 3 trials, including NCT05206773 and NCT05280548 for Fabry Disease, and NCT05222906 for Gaucher Disease Type 3. A Phase 2 trial, NCT02843035, evaluates Venglustat in combination with Cerezyme in Gaucher Disease Type 3.
Yes, Venglustat is also known as GZ402671. Both names refer to the same investigational drug developed by Sanofi for the treatment of lysosomal storage disorders such as Gaucher and Fabry diseases.