Venglustat
An investigational treatment for Gaucher Disease.
The same compound appears under different names depending on the context. Here is how to identify Venglustat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Venglustat
- Development codes
- GZ402671, ibiglustat
- Drug class
- Substrate reduction therapy
- Manufacturer
- Sanofi
- How it's taken
- Taken as an oral tablet once daily.
An investigational oral substrate reduction therapy designed to cross the blood-brain barrier, under FDA priority review for Gaucher disease type 3 with a decision due November 25, 2026.
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How Venglustat works
Venglustat reduces production of glucocerebroside by inhibiting glucosylceramide synthase, similar to eliglustat. The critical difference is that venglustat is designed to cross the blood-brain barrier, so it could reduce the lipid buildup in the brain that causes neurological symptoms in Gaucher type 3, where enzyme infusions cannot reach.
Mechanism: Oral substrate reduction therapy that crosses the blood-brain barrier
Side effects and safety
Clinical trial data reports nausea, diarrhea, and headache as common side effects. Neuropsychiatric effects are being monitored. Full safety profile will be established with Phase 3 completion.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Venglustat
Taken as an oral tablet once daily. The brain-penetrant property makes it the first potential treatment for neurological Gaucher disease.
Clinical trial results
The LEAP2MONO Phase 3 trial in Type 3 Gaucher disease (43 patients randomized) met both primary endpoints and 3 of 4 key secondary endpoints. The FDA granted Breakthrough Therapy designation in March 2026, accepted Sanofi's application for priority review in May 2026, and set a decision date of November 25, 2026.
Main registered trial: NCT05222906 on ClinicalTrials.gov. Check it for the current status, sites and contacts before asking about enrollment.
Development history
Developed by Sanofi as a next-generation substrate reduction therapy specifically designed to cross the blood-brain barrier. If approved, Sanofi says it would be the first US treatment for the neurological manifestations of Gaucher disease type 3. An earlier brain-penetrant pill, miglustat, did not help the neurological symptoms of type 3 in a 2008 trial.
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Common questions about Venglustat
▸What is Venglustat?
An investigational oral substrate reduction therapy designed to cross the blood-brain barrier, under FDA priority review for Gaucher disease type 3 with a decision due November 25, 2026.
▸How does Venglustat work?
Venglustat reduces production of glucocerebroside by inhibiting glucosylceramide synthase, similar to eliglustat. The critical difference is that venglustat is designed to cross the blood-brain barrier, so it could reduce the lipid buildup in the brain that causes neurological symptoms in Gaucher type 3, where enzyme infusions cannot reach.
▸What are the side effects of Venglustat?
Clinical trial data reports nausea, diarrhea, and headache as common side effects. Neuropsychiatric effects are being monitored. Full safety profile will be established with Phase 3 completion.
▸How is Venglustat taken?
Taken as an oral tablet once daily. The brain-penetrant property makes it the first potential treatment for neurological Gaucher disease.
▸Is Venglustat FDA approved?
Venglustat is currently in phase 3 clinical trials for Gaucher Disease. It has not yet received FDA approval.
▸Why is venglustat's ability to cross the blood-brain barrier important?
Current Gaucher treatments cannot reach the brain. Gaucher disease types 2 and 3 involve neurological damage from lipid buildup in brain cells, and venglustat's application covers type 3. Venglustat is designed to cross the blood-brain barrier; miglustat also crosses it but failed to help type 3 neurological symptoms in a 2008 trial.
▸What phase of development is venglustat in?
Venglustat is under FDA priority review for Type 3 Gaucher disease, with a decision date of November 25, 2026. It is not yet approved. The application rests on the LEAP2MONO Phase 3 trial, which met both primary endpoints.
▸How does venglustat compare to eliglustat?
Both are oral substrate reduction therapies that inhibit glucosylceramide synthase, but venglustat can cross the blood-brain barrier while eliglustat cannot. That is why it is being developed for the neurological symptoms of type 3, which eliglustat does not address.
▸What side effects have been reported in clinical trials?
Nausea, diarrhea, and headache are the most commonly reported side effects in clinical trials. Neuropsychiatric effects are being monitored. The full safety profile will be established with Phase 3 completion and regulatory review.
▸How is venglustat administered?
Venglustat is taken as an oral tablet once daily, which would make it convenient for long-term use in Gaucher disease management if approved.
▸What development codes is venglustat known by?
Venglustat has been developed under the codes GZ402671 (Sanofi designation) and ibiglustat (earlier investigational name).
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Sanofi · 2026-05-28. Sanofi's venglustat accepted for priority review in the US to treat type 3 Gaucher disease. https://www.sanofi.com/en/media-room/press-releases/2026/2026-05-28-05-00-00-3302498
- Sanofi · 2026-03-18. Sanofi's venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher disease. https://www.sanofi.com/en/media-room/press-releases/2026/2026-03-18-06-00-00-3257888
- ClinicalTrials.gov. LEAP2MONO: venglustat in type 3 Gaucher disease (Phase 3). https://clinicaltrials.gov/study/NCT05222906