The Wilson’s Disease market report also offers comprehensive insights into the Wilson’s Disease market size, share, Wilson’s Disease epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Wilson’s Disease market size growth forward.
Some of the key highlights from the Wilson’s Disease Market Insights Report:
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Several key pharmaceutical companies, including Orphalan, AstraZeneca,Vivet Therapeutics,Pfizer,Ultragenyx Pharmaceutical, and others, are developing novel products to improve the Wilson’s Disease treatment outlook.
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In January 2025, Eton Pharmaceuticals, Inc., announced that Galzin is an FDA-approved treatment for Wilson Disease, an ultra-rare metabolic disorder.
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According to DelveInsight’s estimates, there were approximately 8,000 diagnosed prevalent cases of Wilson’s disease across the 7MM in 2023. These cases are expected to rise during the forecast period (2024–2034).
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In 2023, the market for Wilson’s Disease was the largest in the US within the 7MM, valued at around USD 126 million, with expectations for a 13% compound annual growth rate (CAGR) over the forecast period.
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Current treatment options primarily include Chelating agents (D-penicillamine, trientine), Zinc Salts, and others. Chelating agents accounted for the largest share of the market, reaching approximately USD 123 million in 2023 for Wilson’s Disease treatment in the US.
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In May 2022, the US FDA approved Orphalan’s CUVRIOR (trientine tetrahydrochloride) for treating adults with stable Wilson’s disease who are decoupled and tolerant to penicillamine.
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The Wilson’s Disease treatment market in the 7MM is driven by factors such as increasing awareness, early diagnosis, advances in genetic testing, and ongoing research into new therapies. Rising prevalence, along with greater healthcare investments in rare diseases, further contributes to market growth. Additionally, patient advocacy efforts are helping to improve treatment options and expand the market.
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The US FDA has also granted Fast Track designation to VTX-801, a gene therapy from Vivet currently in clinical development for Wilson’s Disease. This designation is part of the FDA’s Fast Track program, aimed at expediting the development and review of innovative treatments for serious conditions with unmet medical needs.
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As per DelveInsight analysis, the Wilson’s Disease market is anticipated to witness growth at a considerable CAGR
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Wilson’s Disease Overview
According to the European Association for the Study of the Liver (EASL), Wilson’s disease is a genetic disorder characterized by impaired biliary copper excretion, leading to its accumulation, particularly in the liver and brain.
The condition is progressive, and if untreated, it can result in liver disease, central nervous system dysfunction, and even death. Early detection and treatment can help prevent severe long-term disability and life-threatening complications. Wilson’s disease typically presents with a combination of liver, neurological, and psychiatric symptoms. In children and young adults, liver disease is often the first sign, whereas individuals diagnosed later in life may not exhibit obvious liver symptoms, though mild liver disease may still be present. Symptoms of liver disease include jaundice (yellowing of the skin or eyes), fatigue, loss of appetite, and abdominal swelling. In adults, neurological or psychiatric symptoms are often the first to appear, and they are common in young adults with the condition. These symptoms may include clumsiness, tremors, difficulty walking, speech issues, cognitive impairment, depression, anxiety, and mood swings. Wilson’s disease is caused by a mutation in the ATP7B gene, which is responsible for copper transport. The disease occurs when an individual inherits the mutated gene from both parents.
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Wilson’s Disease Epidemiology Insights
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In 2023, there were around 37,000 prevalent cases of Wilson’s Disease across the 7MM, yet the treatment market for the condition lacks therapies specifically approved for its management. According to the analysis, the United States recorded the highest number of diagnosed prevalent cases, with approximately 2,600, followed by Japan with nearly 2,200 cases. Conversely, Spain had the fewest diagnosed prevalent cases, totaling approximately 300.
Wilson’s Disease Epidemiology Segmentation
DelveInsight’s Wilson’s Disease market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Wilson’s Disease historical patient pools and forecasted Wilson’s Disease patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Wilson’s Disease Market report proffers epidemiological analysis for the study period 2020-34 in the 7MM segmented into:
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Wilson’s Disease Prevalence
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Age-Specific Wilson’s Disease Prevalence
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Gender-Specific Wilson’s Disease Prevalence
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Diagnosed and Treatable Cases of Wilson’s Disease
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Wilson’s Disease Market Insights:
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In 2023, the therapeutic market for Wilson’s Disease in the 7MM was valued at approximately USD 295 million. The market is projected to grow at a CAGR of 12%, driven by increased disease awareness, improved diagnostic capabilities, and the introduction of emerging therapies.
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Among the 7MM, the United States represented the largest market share, accounting for roughly 43% of the total market in 2023. This was significantly higher compared to other major markets, including the EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
Wilson’s Disease Market Outlook
Several medications are available for treating Wilson’s disease, including D-penicillamine, trientine, zinc, tetrathiomolybdate, and dimercaprol. Once diagnosed, lifelong treatment is necessary.
Treatment options fall into three categories:
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Chelating agents: These medications remove copper from the body via urinary excretion. Examples include penicillamine (CUPRIMINE) and trientine dihydrochloride (SYPRINE).
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Zinc salts: These prevent the absorption of dietary copper in the gut. Zinc acetate is preferred due to its superior gastric tolerance.
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Tetrathiomolybdate: This dual-action drug blocks copper absorption and binds toxic copper in the bloodstream, rendering it harmless.
Key Treatment Details:
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Trientine: Developed in 1969 as an alternative for patients intolerant to D-penicillamine, trientine promotes copper excretion through urine. The recommended dose ranges from 750–1500 mg/day in divided doses, with a maintenance dose of 750–1000 mg/day.
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D-penicillamine: Introduced in 1956 as the first oral treatment, it chelates copper and facilitates its excretion via urine. Co-administration with pyridoxine (25–50 mg daily) is recommended to prevent vitamin B6 deficiency. Hepatic function typically improves within 2–6 months. Dosage begins at 250–500 mg/day, increasing by 250 mg every 4–7 days to a maximum of 1–1.5 g/day, divided into multiple doses.
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Zinc salts: First used in the 1960s, zinc salts (like zinc acetate) promote copper elimination through feces by inducing metallothionein in enterocytes. This helps normalize copper levels in the blood.
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Tetrathiomolybdate (TTM): Used in Europe since 2008, TTM promotes copper excretion via urine and feces and is particularly effective for severe neurological symptoms. Common side effects include elevated liver enzymes and bone marrow suppression. TTM is not yet FDA-approved in the United States.
Emerging Treatments:
Several new drugs are under development for Wilson’s disease, including VTX-801 by Vivet Therapeutics and UX701 by Ultragenyx Pharmaceutical. These agents aim to offer improved treatment options for this challenging condition.
Wilson’s Disease Marketed Drugs
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CUVRIOR/CUPRIOR (trientine tetrahydrochloride): Orphalan
Wilson’s Disease Emerging Drugs
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VTX-801: Vivet Therapeutics
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UX701: Ultragenyx Pharmaceutical
Wilson’s Disease Key Companies
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Orphalan, AstraZeneca,Vivet Therapeutics,Pfizer,Ultragenyx Pharmaceutical, and others
For more information, visit Wilson’s Disease Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Wilson’s Disease Market Report:
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11 Years Forecast
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7MM Coverage
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Descriptive overview of Wilson’s Disease, causes, signs and symptoms, diagnosis, treatment
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Comprehensive insight into Wilson’s Disease epidemiology in the 7MM
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Wilson’s Disease marketed and emerging therapies
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Wilson’s Disease companies
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Wilson’s Disease market drivers and barriers
Table of Contents:
1 Wilson’s Disease Market Key Comprehensive Insights
2 Wilson’s Disease Market Report Introduction
3 Competitive Intelligence Analysis for Wilson’s Disease
4 Wilson’s Disease Market Analysis Overview at a Glance
5 Executive Summary of Wilson’s Disease
6 Wilson’s Disease Epidemiology and Market Methodology
7 Wilson’s Disease Epidemiology and Patient Population
8 Wilson’s Disease Patient Journey
9 Wilson’s Disease Treatment Algorithm, Wilson’s Disease Current Treatment, and Medical Practices
10 Key Endpoints in Wilson’s Disease Clinical Trials
11 Wilson’s Disease Marketed Therapies
12 Wilson’s Disease Emerging Therapies
13 Wilson’s Disease: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Wilson’s Disease
16 Wilson’s Disease Market Key Opinion Leaders Reviews
18 Wilson’s Disease Market Drivers
19 Wilson’s Disease Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
Related Reports:
Wilson’s Disease Epidemiology 2034
DelveInsight’s “Wilson’s Disease – Epidemiology Forecast to 2034” report delivers an in-depth understanding of the disease, historical and forecasted Wilson’s Disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Wilson’s Disease Pipeline 2024
“Wilson’s Disease Pipeline Insights, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Wilson’s Disease market. A detailed picture of the Wilson’s Disease pipeline landscape is provided, which includes the disease overview and Wilson’s Disease treatment guidelines.
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