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Wilson Disease

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Pipeline Programs
5
Companies
6
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Orphalan
OrphalanFrance - Paris
2 programs
NCC-Sp AssayN/A1 trial
PenicillaminePHASE_32 trials
Active Trials
NCT05783687Completed50Est. Sep 2025
NCT07465718Not Yet Recruiting38Est. Feb 2028
NCT03539952Completed77Est. Jan 2022
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
ALXN1840N/A
CENTOGENE
CENTOGENEGermany - Rostock
1 program
Establishment of Human Cellular Disease Models for Wilson DiseaseN/A1 trial
Active Trials
NCT03867526Completed40Est. Dec 2019
Bausch Health
Bausch HealthQUEBEC, Quebec, Canada
1 program
Once a day TrientineN/A1 trial
Active Trials
NCT01472874Completed8Est. Jul 2011
Ultragenyx Pharmaceutical
1 program
UX701PHASE_1_21 trial
Active Trials
NCT04884815Active Not Recruiting82Est. Mar 2034

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
OrphalanPenicillamine
OrphalanPenicillamine
Ultragenyx PharmaceuticalUX701
OrphalanNCC-Sp Assay
CENTOGENEEstablishment of Human Cellular Disease Models for Wilson Disease
Bausch HealthOnce a day Trientine

Clinical Trials (6)

Total enrollment: 295 patients across 6 trials

NCT07465718OrphalanPenicillamine

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Start: Jul 2026Est. completion: Feb 202838 patients
Phase 3Not Yet Recruiting
NCT03539952OrphalanPenicillamine

Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease

Start: Sep 2018Est. completion: Jan 202277 patients
Phase 3Completed

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Start: Sep 2021Est. completion: Mar 203482 patients
Phase 1/2Active Not Recruiting

Real World Evidence Study in Subjects With Wilson's Disease

Start: Jun 2023Est. completion: Sep 202550 patients
N/ACompleted
NCT03867526CENTOGENEEstablishment of Human Cellular Disease Models for Wilson Disease

Establishment of Human Cellular Disease Models for Wilson Disease

Start: Jun 2018Est. completion: Dec 201940 patients
N/ACompleted
NCT01472874Bausch HealthOnce a day Trientine

Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

Start: Jan 2010Est. completion: Jul 20118 patients
N/ACompleted

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.