Clinical Trials
Wilson Disease Drug Pipeline Analysis 2024: By Clinical Trial Phase: Late-Stage Products (Phase III and Phase IV), Mid-Stage Products (Phase II), Early-Stage Products (Phase I), Preclinical and Discovery Stage Products; By Drug Class: Bispecific Antibody, Peptides, Small Molecule, Gene Therapy; Route of Administration; Drug Profile; Key Players
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.
Key Takeaways
- Major companies involved in the Wilson disease drugs market include Alexion Pharmaceuticals, Inc., Vivet Therapeutics SAS, Ultragenyx Pharmaceutical Inc. and Nobelpharma, among others.
- Leading drugs currently under pipeline include Copper Transporting ATPase 2, VTX-801 and UX701-CL001 among others.
- Regulatory authorities like the United States FDA and EMA play an essential role in the drug pipeline for Wilson disease as they are offering breakthrough designations and fast-track approvals to various drugs, intended to provide effective treatment to patients suffering from Wilson disease.
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 Drug 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 – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of Wilson disease drugs based on various segmentations such as:
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
By Drug Class
EMR’s pipeline assessment report covers 50+ drug analyses based on drug classes:
- Bispecific Antibody
- Peptides
- Small Molecule
- Gene Therapy
By Route of Administration
EMR’s pipeline assessment report covers 50+ drug analyses based on the route of administration.
- Oral
- Parenteral
- Others
Wilson Disease – Pipeline Assessment Segmentation, By Phases
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 report for the Wilson disease drug pipeline 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
Wilson Disease – Pipeline Drugs Profile
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.
| Scope of the Report | Details |
| Drug Pipeline by Clinical Trial Phase |
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| Route of Administration |
|
| Drug Classes |
|
| Leading Sponsors Covered |
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| Geographies Covered |
|
Key Questions Answered in the Wilson Disease – Pipeline Assessment Report
- What is the current landscape of Wilson disease pipeline drugs?
- How many companies are developing Wilson disease drugs?
- How many phase III and phase IV drugs are currently present in Wilson disease pipeline?
- Which companies/institutions are leading the Wilson disease drug development?
- What is the efficacy and safety profile of Wilson disease pipeline drugs?
- What are the opportunities and challenges present in the Wilson disease drug pipeline landscape?
- Which company is conducting major trials for Wilson disease drugs?
- What are the geographies covered for clinical trials in Wilson disease?
- What are emerging trends in Wilson disease clinical trials?
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.
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
*Complete list of drugs covered will be provided in the report.
The EMR team aims to provide comprehensive coverage of the Top Drugs for each Phase, considering factors such as the company's financial standing, geographic presence, and market position to ensure thorough analysis in this section.
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