VivoSeq
Rare Disease

Gaucher Disease

31

Pipeline Programs↗

7

Active Trials↗

14

Companies Involved↗

Gaucher Disease is a moderately active landscape with 31 programs across 14 companies. The most active players are Roche, Prism Pharmaceuticals, Boehringer Ingelheim. 5 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

53/100

Pipeline Velocity

0/100

Mech. Concentration

40/100

Completion Rate

85/100

Attrition Rate

9/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Roche logo

Roche

1 program

Phase 1P

Prism Pharmaceuticals

1 program

Phase 1Boehringer Ingelheim logo

Boehringer Ingelheim

1 program

Phase 2A

Amicus Therapeutics

2 programs

Phase 2G

Genzyme, a Sanofi Company

2 programs

Phase 3Johnson & Johnson logo

Johnson & Johnson

1 program

Phase 3

Pipeline

Programs by Phase

Preclinical1

Velaglucerase Alfa

Takeda

Phase 17

RG6026

Roche

Methylprednisolone

Pfizer

Prednisone

Roxane Laboratories

PRX-112

Protalix

VGN-R08b

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Phase 210

Afegostat Tartrate

Amicus Therapeutics

Venglustat

Sanofi

AVR-RD-02

AVROBIO

Imiglucérase (drug) Pharmacokinetics

University Hospital, Clermont-Ferrand

Glucocerebrosidase

National Center for Research Resources (NCRR)

Phase 38

Miglustat

Johnson & Johnson

Eliglustat

Genzyme, a Sanofi Company

Gene Therapy

Capsida Biotherapeutics, Inc.

Imiglucerase

Genzyme, a Sanofi Company

ISU302

ISU Abxis Co., Ltd.

Approved5

Cyclophosphamide

Baxter Healthcare Corporation

Busulfan

Akero Therapeutics

Cerezyme

Sanofi

Taliglucerase Alfa

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

Elelyso

Shaare Zedek Medical Center

Analysis

Modality Breakdown

82%

Small molecule

27 of 33 classified programs — the dominant modality here.

18%ALL OTHERSProtein / enzyme515%Gene therapy13%

Small molecule leads; the ring shows all 33 classified programs, each share of the total. Modality classified for 97% of 37 pipeline programs.

Analysis

Trial Status

55

clinical trials

Completed35 · 64%
Unknown status4 · 7%
Withdrawn4 · 7%
Recruiting4 · 7%
Active, not recruiting3 · 5%
Terminated1 · 2%
Other4 · 7%

Clinical Trials

Trial Activity

55 trials

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type I
Recruiting·—·N/A·NCT06162338·3 enrolled
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
Recruiting·XXinhua Hospital, Shanghai Jiao Tong University School of Medicine·Phase 1·NCT06272149·6 enrolled
Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)
Recruiting·UUniversity of Minnesota·N/A·NCT02437396·34 enrolled
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Recruiting·Eli Lilly logoEli Lilly·Preclinical·NCT05487599·15 enrolled
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Active, not recruiting·Eli Lilly logoEli Lilly·Phase 1/2·NCT04411654·7 enrolled
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Active, not recruiting·AAgyany Pharma LTD·N/A·NCT07285369·12 enrolled
IMAGINE-1: Miglustat in Niemann-Pick Disease
Active, not recruiting·Sanofi logoSanofi·Phase 3·NCT05700003·80 enrolled
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Completed·Pfizer logoPfizer·Phase 3·NCT01422187·19 enrolled
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Completed·Takeda logoTakeda·Phase 1/2·NCT00391625·10 enrolled
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Completed·Sanofi logoSanofi·Phase 3·NCT03485677·57 enrolled
Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
Completed·UUniversity Research Foundation for Lysosomal Storage Diseases, Inc.·N/A·NCT01136304·173 enrolled
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Completed·AAmicus Therapeutics·Phase 2·NCT00446550·19 enrolled
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Completed·Sanofi logoSanofi·Phase 4·NCT00364858·95 enrolled
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
Completed·Sanofi logoSanofi·Phase 4·NCT04656600·12 enrolled
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Completed·Takeda logoTakeda·Phase 3·NCT00635427·95 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)