Wilson Disease Pipeline Analysis Report 2025
Description
According to the American Liver Foundation, about 1 in 30,000 people have Wilson disease in the United States. In addition, a study conducted in the United Kingdom found that 1 in 7,000 people have gene mutations which cause Wilson disease. There has been a significant emphasis on developing effective treatments to manage the condition effectively.
Report Coverage
The Wilson Disease Drug Pipeline Report by Expert Market Research gives comprehensive insights into Wilson disease drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Wilson disease. The report includes the analysis of over 100 pipeline drugs and 50+ companies. It will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Wilson disease.
The detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing product development activities related to Wilson disease are covered.
Wilson Disease Pipeline Outlook
Wilson disease is a rare genetic disorder where excess copper builds up in the body. This condition affects the brain, liver, eyes, and other organs. Wilson disease can lead to organ damage. Most people with the disorder are diagnosed between the ages of 5-35. The symptoms of Wilson disease vary from person to person and may include tiredness, loss of appetite, yellowing of the skin, fluid buildup in the legs or stomach, speech problems and muscle stiffness among others.
The treatment for Wilson disease includes lowering toxic levels of copper in the body and preventing organ damage. The medicine includes chelating agents, tetrathiomolybdate and penicillamine. People who are suffering from the disease need lifelong treatment.
Companies are developing innovative treatments to manage this condition. For instance, GATEWAY is a gene therapy clinical trial for Wilson Disease. The study attempts to assess the safety and efficacy of a drug called VTX-801 which can eliminate excess copper through the natural route. Several Wilson disease drugs in the pipeline are being developed by key pharmaceutical companies. There is an increase in investment activities to combat the rising fatalities associated with Wilson disease.
Wilson Disease Epidemiology
Wilson’s disease is a rare genetic disorder, with prevalence ranging from 1 in 100,000 to 1 in 3 million people. The condition affects males and females equally and is most commonly diagnosed between the ages of 4 and 40, though cases can occur as early as age 3 or as late as 70. The global incidence is estimated at 10 to 30 million cases. Carrier frequency is around 1 in 90 to 1 in 100 individuals. Populations with a high rate of consanguineous marriages tend to show a higher incidence of the disease.
Wilson Disease – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of Wilson disease drug candidates based on several segmentations, including:
By Phase
EMR’s pipeline assessment report covers 50+ drug analyses based on phase.
EMR’s Wilson disease therapeutic assessment report covers 50+ drug analyses based on drug classes:
EMR’s Wilson disease clinical assessment covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase I covers a major share of the total clinical trials conducted for Wilson disease treatment.
Wilson Disease – Pipeline Assessment Segmentation, By Drug Class
The drug class categories covered under Wilson disease pipeline analysis include bispecific antibodies, peptides, small molecules, and gene therapy. When affected by Wilson disease, patients have a faulty copy of the ATP7B gene. This gene is responsible for transporting excess copper out of the liver. Hence, gene therapy is commonly used to treat the condition in patients. Two gene therapies are currently under trial to treat patients with Wilson disease. Furthermore, Ultragenyx is evaluating UX701, an investigational adeno-associated virus (AAV) vector-based gene therapy. The therapy delivers stable expression of the ATP7B copper transporter. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Wilson disease.
Wilson Disease Clinical Trials Assessment – Competitive Dynamics
The EMR Wilson disease drug pipeline report covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Wilson disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Wilson disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Wilson disease drug candidates.
VTX-801
VTX-801, is currently under phase II, developed by Vivet Therapeutics SAS. The trial study is being conducted to assess the safety and tolerability of single ascending doses of VTX-801. It is a gene therapy administered intravenously to patients suffering from Wilson's Disease
UX701-CL001
Ultragenyx Pharmaceutical Inc. is conducting an investigational study to determine the relevance and appropriateness of outcome assessments including biomarkers in Wilson disease population.
ALXN1840
Alexion Pharmaceuticals, Inc. is developing the drug, which is currently under phase II. The study is investigating the effects of ALXN1840 on copper balance in patients suffering from Wilson disease.
Reasons To Buy This Report
The Wilson Disease Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Wilson disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within Wilson disease pipeline insights.
Key Questions Answered in the Wilson Disease – Pipeline Assessment Report
Wilson’s Disease Market
Hereditary Angioedema Therapeutic Market
Hereditary Orotic Aciduria Treatment Market
Report Coverage
The Wilson Disease Drug Pipeline Report by Expert Market Research gives comprehensive insights into Wilson disease drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Wilson disease. The report includes the analysis of over 100 pipeline drugs and 50+ companies. It will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Wilson disease.
The detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing product development activities related to Wilson disease are covered.
Wilson Disease Pipeline Outlook
Wilson disease is a rare genetic disorder where excess copper builds up in the body. This condition affects the brain, liver, eyes, and other organs. Wilson disease can lead to organ damage. Most people with the disorder are diagnosed between the ages of 5-35. The symptoms of Wilson disease vary from person to person and may include tiredness, loss of appetite, yellowing of the skin, fluid buildup in the legs or stomach, speech problems and muscle stiffness among others.
The treatment for Wilson disease includes lowering toxic levels of copper in the body and preventing organ damage. The medicine includes chelating agents, tetrathiomolybdate and penicillamine. People who are suffering from the disease need lifelong treatment.
Companies are developing innovative treatments to manage this condition. For instance, GATEWAY is a gene therapy clinical trial for Wilson Disease. The study attempts to assess the safety and efficacy of a drug called VTX-801 which can eliminate excess copper through the natural route. Several Wilson disease drugs in the pipeline are being developed by key pharmaceutical companies. There is an increase in investment activities to combat the rising fatalities associated with Wilson disease.
Wilson Disease Epidemiology
Wilson’s disease is a rare genetic disorder, with prevalence ranging from 1 in 100,000 to 1 in 3 million people. The condition affects males and females equally and is most commonly diagnosed between the ages of 4 and 40, though cases can occur as early as age 3 or as late as 70. The global incidence is estimated at 10 to 30 million cases. Carrier frequency is around 1 in 90 to 1 in 100 individuals. Populations with a high rate of consanguineous marriages tend to show a higher incidence of the disease.
Wilson Disease – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of Wilson disease drug candidates based on several segmentations, including:
By Phase
EMR’s pipeline assessment report covers 50+ drug analyses based on phase.
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
EMR’s Wilson disease therapeutic assessment report covers 50+ drug analyses based on drug classes:
- Bispecific Antibody
- Peptides
- Small Molecule
- Gene Therapy
EMR’s Wilson disease clinical assessment covers 50+ drug analyses based on the route of administration.
- Oral
- Parenteral
- Others
The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase I covers a major share of the total clinical trials conducted for Wilson disease treatment.
Wilson Disease – Pipeline Assessment Segmentation, By Drug Class
The drug class categories covered under Wilson disease pipeline analysis include bispecific antibodies, peptides, small molecules, and gene therapy. When affected by Wilson disease, patients have a faulty copy of the ATP7B gene. This gene is responsible for transporting excess copper out of the liver. Hence, gene therapy is commonly used to treat the condition in patients. Two gene therapies are currently under trial to treat patients with Wilson disease. Furthermore, Ultragenyx is evaluating UX701, an investigational adeno-associated virus (AAV) vector-based gene therapy. The therapy delivers stable expression of the ATP7B copper transporter. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Wilson disease.
Wilson Disease Clinical Trials Assessment – Competitive Dynamics
The EMR Wilson disease drug pipeline report covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Wilson disease clinical trials:
- Vivet Therapeutics SAS
- Alexion Pharmaceuticals, Inc.
- Ultragenyx Pharmaceutical Inc
- Nobelpharma Co. Ltd.
- Others
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Wilson disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Wilson disease drug candidates.
VTX-801
VTX-801, is currently under phase II, developed by Vivet Therapeutics SAS. The trial study is being conducted to assess the safety and tolerability of single ascending doses of VTX-801. It is a gene therapy administered intravenously to patients suffering from Wilson's Disease
UX701-CL001
Ultragenyx Pharmaceutical Inc. is conducting an investigational study to determine the relevance and appropriateness of outcome assessments including biomarkers in Wilson disease population.
ALXN1840
Alexion Pharmaceuticals, Inc. is developing the drug, which is currently under phase II. The study is investigating the effects of ALXN1840 on copper balance in patients suffering from Wilson disease.
Reasons To Buy This Report
The Wilson Disease Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Wilson disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within Wilson disease pipeline insights.
Key Questions Answered in the Wilson Disease – Pipeline Assessment Report
- Which companies/institutions are leading the Wilson disease drug development?
- What is the efficacy and safety profile of Wilson disease pipeline drugs?
- Which company is leading the Wilson disease pipeline development activities?
- What is the current Wilson disease commercial assessment?
- What are the opportunities and challenges present in the Wilson disease drug pipeline landscape?
- What is the efficacy and safety profile of Wilson disease pipeline drugs?
- Which company is conducting major trials for Wilson disease drugs?
- Which companies/institutions are involved in Wilson disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in Wilson disease?
Wilson’s Disease Market
Hereditary Angioedema Therapeutic Market
Hereditary Orotic Aciduria Treatment Market
Table of Contents
200 Pages
- 1 Preface
- 1.1 Introduction
- 1.2 Objectives of the Study
- 1.3 Research Methodology and Assumptions
- 2 Executive Summary
- 3 Overview of Wilson Disease
- 3.1 Signs and Symptoms
- 3.2 Causes
- 3.3 Risk Factors
- 3.4 Diagnosis
- 3.5 Treatment
- 4 Patient Profile
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Treatment Success Rate
- 5 Wilson Disease: Epidemiology Snapshot
- 5.1 Wilson Disease Incidence by Key Markets
- 5.2 Wilson Disease– Patients Seeking Treatment in Key Markets
- 6 Wilson Disease: Market Dynamics
- 6.1 Market Drivers and Constraints
- 6.2 SWOT Analysis
- 7 Wilson Disease: Key Facts Covered
- 7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
- 7.2 Top Countries Contributing to Clinical Trials in Europe
- 7.3 Top Countries Contributing to Clinical Trials in North America
- 7.4 Top Countries Contributing to Clinical Trials in Other Regions
- 8 Wilson Disease, Drug Pipeline Assessment
- 8.1 Assessment by Treatment Type
- 8.2 Assessment by Route of Administration
- 8.3 Assessment by Drug Class
- 9 EMR Drug Pipeline Comparative Analysis
- 9.1 List of Wilson Disease Pipeline Drugs
- 9.1.1 By Company
- 9.1.2 By Phase
- 9.1.3 By Indication
- 9.1.4 By Trial Status
- 9.1.5 By Funder Type
- 9.2 EMR Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
- 10 Wilson Disease Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
- 10.1 Comparative Analysis for Late-Stage Drugs
- 10.1.1 Study Type
- 10.1.2 Recruitment Status
- 10.1.3 Company
- 10.1.4 Funder Type
- 10.2 Product Level Analysis*
- 10.2.1 ALXN1840
- 10.2.1.1 Product Description
- 10.2.1.2 Trial ID
- 10.2.1.3 Sponsor Name
- 10.2.1.4 Study Type
- 10.2.1.5 Drug Class
- 10.2.1.6 Eligibility Criteria
- 10.2.1.7 Study Record Dates
- 10.2.1.7.1 First Submitted
- 10.2.1.7.2 First Posted
- 10.2.1.7.3 Last Update Posted
- 10.2.1.7.4 Last Verified
- 10.2.1.8 Indication
- 10.2.1.9 Study Design
- 10.2.1.10 Recruitment Status
- 10.2.1.11 Enrollment (Estimated)
- 10.2.1.12 Location Countries
- 10.2.1.13 Recent Results
- 10.2.2 NPC-02
- 10.2.3 Trientine Dihydrochloride
- 10.2.4 Other Drug
- 11 Wilson Disease Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
- 11.1 Comparative Analysis for Mid-Stage Drugs
- 11.1.1 Study Type
- 11.1.2 Recruitment Status
- 11.1.3 Company
- 11.1.4 Funder Type
- 11.2 Product Level Analysis*
- 11.2.1 VTX-801
- 11.2.1.1 Product Description
- 11.2.1.2 Trial ID
- 11.2.1.3 Sponsor Name
- 11.2.1.4 Study Type
- 11.2.1.5 Drug Class
- 11.2.1.6 Eligibility Criteria
- 11.2.1.7 Study Record Dates
- 11.2.1.7.1 First Submitted
- 11.2.1.7.2 First Posted
- 11.2.1.7.3 Last Update Posted
- 11.2.1.7.4 Last Verified
- 11.2.1.8 Indication
- 11.2.1.9 Study Design
- 11.2.1.10 Recruitment Status
- 11.2.1.11 Enrollment (Estimated)
- 11.2.1.12 Location Countries
- 11.2.1.13 Recent Results
- 11.2.2 UX701
- 11.2.3 Bis-Choline Tetrathiomolybdate
- 11.2.4 Other Drugs
- 12 Wilson Disease Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
- 12.1 Comparative Analysis for Early-Stage Drugs
- 12.1.1 Study Type
- 12.1.2 Recruitment Status
- 12.1.3 Company
- 12.1.4 Funder Type
- 12.2 Product Level Analysis*
- 12.2.1 VTX-801
- 12.2.1.1 Product Description
- 12.2.1.2 Trial ID
- 12.2.1.3 Sponsor Name
- 12.2.1.4 Study Type
- 12.2.1.5 Drug Class
- 12.2.1.6 Eligibility Criteria
- 12.2.1.7 Study Record Dates
- 12.2.1.7.1 First Submitted
- 12.2.1.7.2 First Posted
- 12.2.1.7.3 Last Update Posted
- 12.2.1.7.4 Last Verified
- 12.2.1.8 Indication
- 12.2.1.9 Study Design
- 12.2.1.10 Recruitment Status
- 12.2.1.11 Enrollment (Estimated)
- 12.2.1.12 Location Countries
- 12.2.2 UX701
- 12.2.3 ALXN1840
- 12.2.4 Other Drugs
- 13 Wilson Disease Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
- 13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
- 13.1.1 Study Type
- 13.1.2 Recruitment Status
- 13.1.3 Company
- 13.1.4 Funder Type
- 13.2 Product Level Analysis*
- 13.2.1 Drug 1
- 13.2.1.1 Product Description
- 13.2.1.2 Trial ID
- 13.2.1.3 Sponsor Name
- 13.2.1.4 Study Type
- 13.2.1.5 Drug Class
- 13.2.1.6 Eligibility Criteria
- 13.2.1.7 Study Record Dates
- 13.2.1.7.1 First Submitted
- 13.2.1.7.2 First Posted
- 13.2.1.7.3 Last Update Posted
- 13.2.1.7.4 Last Verified
- 13.2.1.8 Indication
- 13.2.1.9 Study Design
- 13.2.1.10 Recruitment Status
- 13.2.1.11 Enrollment (Estimated)
- 13.2.1.12 Location Countries
- 13.2.2 Drug 2
- 13.2.3 Other Drugs
- 14 Wilson Disease, Key Drug Pipeline Companies
- 14.1 Vivet Therapeutics SAS
- 14.1.1 Company Snapshot
- 14.1.2 Pipeline Product Portfolio
- 14.1.3 Financial Analysis
- 14.1.4 Recent News and Developments
- 14.2 Alexion Pharmaceuticals, Inc.
- 14.2.1 Company Snapshot
- 14.2.2 Pipeline Product Portfolio
- 14.2.3 Financial Analysis
- 14.2.4 Recent News and Developments
- 14.3 Ultragenyx Pharmaceutical Inc
- 14.3.1 Company Snapshot
- 14.3.2 Pipeline Product Portfolio
- 14.3.3 Financial Analysis
- 14.3.4 Recent News and Developments
- 14.4 Nobelpharma Co. Ltd.
- 14.4.1 Company Snapshot
- 14.4.2 Pipeline Product Portfolio
- 14.4.3 Financial Analysis
- 14.4.4 Recent News and Developments
- 15 Regulatory Framework for Drug Approval, By Region
- 16 Terminated or Suspended Pipeline Products
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