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Venglustat

Phase 3

Fabry Disease | Small molecule | Metabolic |Sanofi|Last Updated: Mar 10, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment226
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05280548A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry DiseasePHASE3 ACTIVE NOT_RECRUITING 104 — — May 3, 2022Dec 6, 2027Mar 10, 202654 United States, Austria +17
NCT05206773A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry DiseasePHASE3 ACTIVE NOT_RECRUITING 122 — — Mar 11, 2022Jan 4, 2027Feb 4, 202658 United States, Argentina +19
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Study Endpoints
Primary Endpoints
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
from baseline to 18 months
Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain)
From baseline to 6 months
Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain)
From baseline to 12 months
Secondary Endpoints
Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation
from baseline to 18 months
Change in T1 relaxation time, measured by cardiac MRI (central reading)
from baseline to 18 months
Change in global longitudinal strain, measured by echocardiography (central reading)
from baseline to 18 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VenglustatEXPERIMENTALParticipants will receive venglustat once daily, orally
Standard of Care TherapyACTIVE_COMPARATORParticipants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information)
PlaceboPLACEBO_COMPARATORParticipants will receive placebo once daily for 12 months
Interventions
NameTypeDescription
Venglustat (GZ402671)DRUGTablet; Oral
Agalsidase alfaDRUGConcentrate for solution for infusion; IV infusion
Agalsidase beta (GZ419828)DRUGPowder for concentrate for solution for infusion; IV infusion
MigalastatDRUGHard capsules; Oral
PlaceboDRUGPharmaceutical form: Tablet Route of administration: Oral
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites54

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...

Countries:United StatesAustriaCanadaChinaCzechiaDenmarkFranceGermanyGreeceItalyJapanNetherlandsNorwayPolandSouth KoreaSpainTaiwanTurkey (Türkiye)United KingdomArgentinaAustraliaBrazilFinlandMexicoRomaniaSwitzerland
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05206773primaryCompletionDate: changed
LOWMay 26, 2026NCT05280548primaryCompletionDate: changed
LOWMay 24, 2026NCT05206773studyFirstPostDate: changed
LOWMay 24, 2026NCT05280548studyFirstPostDate: changed