Gaucher Disease, Type 1

Rare Diseases
7
Pipeline Programs
3
Companies
10
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
2
0
3
2
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

U
ELIGLUSTAT TARTRATEApproved
eliglustat tartrate
Unknown Company
oral2024

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Sanofi
SanofiPARIS, France
4 programs
1
1
1
CerezymePhase 44 trials
Eliglustat tartratePhase 31 trial
Eliglustat tartratePhase 21 trial
A Screening Study Evaluating Disease Status of Gaucher Type I PatientsN/A1 trial
Active Trials
NCT00795197Withdrawn0Est. Dec 2009
NCT00358150Completed26Est. Dec 2015
NCT00943111Completed160Est. Jun 2015
+4 more trials
Takeda
TakedaTOKYO, Japan
2 programs
2
VPRIV ®,Phase 31 trial
VPRIV®Phase 31 trial
Active Trials
NCT00430625Completed25Est. Apr 2009
NCT00635427Completed95Est. Dec 2012
Amicus Therapeutics
Amicus TherapeuticsPA - Philadelphia
1 program
1
Afegostat tartratePhase 21 trial
Active Trials
NCT00433147CompletedEst. Feb 2008

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
SanofiCerezyme
SanofiCerezyme
SanofiCerezyme
SanofiEliglustat tartrate
TakedaVPRIV®
TakedaVPRIV ®,
SanofiCerezyme
Amicus TherapeuticsAfegostat tartrate
SanofiEliglustat tartrate
SanofiA Screening Study Evaluating Disease Status of Gaucher Type I Patients

Clinical Trials (10)

Total enrollment: 465 patients across 10 trials

NCT04656600SanofiCerezyme

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

Start: Mar 2021Est. completion: Oct 202312 patients
Phase 4Completed
NCT00364858SanofiCerezyme

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Start: Dec 2001Est. completion: Feb 200795 patients
Phase 4Completed
NCT00365131SanofiCerezyme

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Start: Dec 1997Est. completion: Jul 200440 patients
Phase 4Completed
NCT00943111SanofiEliglustat tartrate

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Start: Sep 2009Est. completion: Jun 2015160 patients
Phase 3Completed
NCT00635427TakedaVPRIV®

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Start: Mar 2008Est. completion: Dec 201295 patients
Phase 3Completed
NCT00430625TakedaVPRIV ®,

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

Start: Feb 2007Est. completion: Apr 200925 patients
Phase 3Completed
NCT02843035SanofiCerezyme

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Start: Jan 2017Est. completion: Oct 202612 patients
Phase 2Active Not Recruiting
NCT00433147Amicus TherapeuticsAfegostat tartrate

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

Start: Mar 2007Est. completion: Feb 2008
Phase 2Completed
NCT00358150SanofiEliglustat tartrate

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Start: Jun 2006Est. completion: Dec 201526 patients
Phase 2Completed
NCT00795197SanofiA Screening Study Evaluating Disease Status of Gaucher Type I Patients

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Start: Dec 2008Est. completion: Dec 20090
N/AWithdrawn

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space

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