Wilson Disease

3
Pipeline Programs
4
Companies
6
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
1
0
0
1
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

U
PENICILLAMINEApproved
penicillamine
Unknown Company
Antirheumatic Agent [EPC]oral2021

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Orphalan
OrphalanFrance - Paris
2 programs
1
PenicillaminePhase 32 trials
NCC-Sp AssayN/A1 trial
Active Trials
NCT05783687Completed50Est. Sep 2025
NCT07465718Not Yet Recruiting38Est. Jan 2028
NCT03539952Completed77Est. Jan 2022
Ultragenyx Pharmaceutical
1 program
1
UX701Phase 1/21 trial
Active Trials
NCT04884815Active Not Recruiting82Est. Mar 2034
C
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
NCT01472874CompletedEst. Jul 2011

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: 287 patients across 6 trials

NCT07465718OrphalanPenicillamine

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

Start: May 2026Est. completion: Jan 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
NCT04884815Ultragenyx PharmaceuticalUX701

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
NCT05783687OrphalanNCC-Sp Assay

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 2011
N/ACompleted

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

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