Venglustat

Pipeline

Status by Indication[3][1]

Competition

Competitive Landscape

Fabry DiseaseGaucher Disease Type 1

11 competitors in Fabry Disease

#DrugCompanyPhaseTrials

1

ELFABRIO

Chiesi Farmaceutici S.p.A.

Phase 3

62

Replagal

Takeda

Phase 3

113

Venglustat

Sanofi

Phase 3

24

Migalastat Hcl

AAmicus Therapeutics

Phase 3

145

Lucerastat

Idorsia Pharmaceuticals Ltd.

Phase 3

56

PRX-102 /kg

Chiesi Farmaceutici S.p.A.

Phase 3

27

Zemplar

AbbVie

Preclinical

18

Biological Data

UUniversity Hospital, Bordeaux

Preclinical

1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Clinical

Trials by Indication[1]

Adult3 trials

A Study of the Safety, Tolerability, and Bioavailability of Orally Administered Venglustat in Healthy Adult Participants

Completed·Sanofi·Phase 1·NCT06418620·9 enrolled

A Study of the Safety, Tolerability, and Bioequivalence of Orally Administered Venglustat in Healthy Adult Participants

Completed·Sanofi·Phase 1·NCT06418607·65 enrolled

A Study of the Safety, Tolerability, and Pharmacokinetics of Orally Administered Venglustat and Itraconazole in Healthy Adult Male Participants

Completed·Sanofi·Phase 1·NCT06421714·8 enrolled

Fabry Disease2 trials

A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months

Active, not recruiting·Sanofi·Phase 3·NCT05206773·122 enrolled

A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy

Active, not recruiting·Sanofi·Phase 3·NCT05280548·104 enrolled

Metabolism1 trial

Excretion Balance, Pharmacokinetics, and Metabolism Following of [14C]-Venglustat Administration in Healthy Male Subjects

Completed·Sanofi·Phase 1·NCT05238714·6 enrolled

Renal Impairment1 trial

Effect of Venglustat in Patients With Renal Impairment

Completed·Sanofi·Phase 1·NCT03687554·24 enrolled

Hepatic Impairment1 trial

A Study in Adults to Investigate the Impact of Mild, Moderate, and Severe Hepatic Impairment on Pharmacokinetics of Venglustat Compared to Participants With Normal Hepatic Function

Completed·Sanofi·Phase 1·NCT05718258·26 enrolled

6 more indications

Autosomal Dominant Polycystic Kidney Disease1 trial

Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat

Terminated·Sanofi·Phase 3·NCT04705051·24 enrolled

Parkinson's Disease1 trial

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

Terminated·Sanofi·Phase 2·NCT02906020·273 enrolled

Treatment1 trial

A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

Terminated·Sanofi·Phase 2/3·NCT03523728·478 enrolled

Double-blind1 trial

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Terminated·Sanofi·Phase 3·NCT04221451·75 enrolled

Gaucher's Disease Type III1 trial

A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT)

Active, not recruiting·Sanofi·Phase 3·NCT05222906·43 enrolled

Gaucher disease type 11 trial

A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Active, not recruiting·Sanofi·Phase 2·NCT02843035·12 enrolled

Details

Drug Info

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