Wilson Disease - Market Insight, Epidemiology and Market Forecast - 2032

This ‘Wilson's Disease (WD) - Market Insights, Epidemiology, and Market Forecast - 2032' report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of WD in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The WD market report provides current treatment practices, emerging drugs, market share of the individual therapies, and current and forecasted Wilson's disease market size from 2019 to 2032, segmented by seven major markets. The report also covers the current Wilson's disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2019-2032

Wilson's disease (WD): Disease Understanding and Treatment Algorithm

Wilson's disease (WD) Overview

According to the European Association for the study of the Liver (EASL), Wilson's disease (WD) is an inherited disorder in which defective biliary excretion of copper leads to its accumulation, particularly in the liver and brain.

The disease is progressive and, if left untreated, may cause liver (hepatic) disease, central nervous system dysfunction, and death. Early diagnosis and treatment may prevent serious long-term disability and life-threatening complications. The features of this condition include a combination of liver disease and neurological and psychiatric problems. Liver disease is typically the initial feature of WD in affected children and young adults; individuals diagnosed at an older age usually do not have symptoms of liver problems, although they may have very mild liver disease. The signs and symptoms of liver disease include yellowing of the skin or whites of the eyes (jaundice), fatigue, loss of appetite, and abdominal swelling. Nervous system or psychiatric problems are often the initial features in individuals diagnosed in adulthood and commonly occur in young adults with WD. Signs and symptoms of these problems can include clumsiness, tremors, difficulty walking, speech problems, impaired thinking ability, depression, anxiety, and mood swings. A mutation in the ATP7B gene, which codes for copper transportation, causes WD. A person must inherit the gene from both parents to have WD.

According to the National Organization of Rare Diseases, WD is a disorder that affects males and females in equal numbers. The disease is found in all races and ethnic groups. Although estimates vary, it is believed that WD occurs in approximately one in 30,000-40,000 people worldwide. Approximately 1 in 90 people may be carriers of the disease gene. Although only about 2,000-3,000 cases have been diagnosed in the United States, other affected individuals may be misdiagnosed with neurological, liver, or psychiatric disorders. According to one estimate, there may be 9,000 people affected by WD in the United States.

WD can affect many different systems of the body. Affected people often develop signs and symptoms of chronic liver disease in their teenage years or early twenties. These features may include jaundice; abnormal fluid retention, which can lead to swelling of the legs and/or abdomen; weight loss; nausea and vomiting, and/or fatigue. Unfortunately, some people may not experience any signs until they suddenly develop acute liver failure.

Affected people often experience a variety of neurologic (central nervous system-related) signs and symptoms, as well. Neurologic features often develop after the liver has retained a significant amount of copper; however, they have been seen in people with little to no liver damage. These symptoms may include tremors, muscle stiffness, and problems with speech, swallowing, and/or physical coordination. Almost all people with neurologic symptoms have Kayser-Fleisher rings - a rusty brown ring around the cornea of the eye that can best be viewed using an ophthalmologist's slit lamp.

Treatment for WD is life-long and aimed at lowering copper levels to nontoxic levels, and at preventing the progression of the disease, and trying to reverse any signs and symptoms that have appeared because of copper accumulation in the body. Treatment may be divided into three parts: first, treatment of symptomatic patients; second, maintenance therapy after the copper has been reduced in affected tissues; and third, in asymptomatic patients, maintenance therapy may be used from the beginning.

The several promising novel therapies, including chelators targeting specific cell types and cell-based and gene therapies, may revolutionize the care for WD patients. However, timely clinical diagnosis remains the main challenge, and many unmet needs exist because of possible clinical deterioration in treated patients.

Wilson's disease (WD) Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnoses available in Wilson's disease (WD) market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

Wilson's disease (WD) Epidemiology

The Wilson's disease (WD) epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The report also provides the diagnosed incident patient pool, trends, and assumptions.

Key Findings

The disease epidemiology covered in the report provides historical and forecasted Wilson's disease (WD) epidemiology segmented as the prevalent cases of Wilson's disease, diagnosed cases of Wilson's disease, prevalent cases of Wilson's disease based on clinical manifestation, and prevalent cases of Wilson's disease based on symptoms. The report includes the diagnosed prevalent scenario of Wilson's disease (WD) symptoms in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise, Wilson's disease (WD) Epidemiology

The epidemiology segment also provides Wilson's disease (WD) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The total prevalent patient population of Wilson's disease (WD) in the 7MM countries was estimated to be about 37,000 cases in 2021.

Wilson's disease (WD) Drug Chapters

The drug chapter segment of the Wilson's disease (WD) report encloses the detailed analysis of Wilson's disease (WD) marketed drugs and late stage (Phase-III, Phase-II/III, Phase-II, and Phase-I/II) pipeline drugs. It also helps understand Wilson's disease (WD) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

A number of drugs are available to treat WD, including D-penicillamine, trientine, zinc, tetrathiomolybdate, and dimercaprol. Once the diagnosis has been made, treatment needs to be lifelong. Treatment for WD includes three types of medications. First, those that remove (chelate) copper from the body by urinary excretion, such as penicillamine (Cuprimine) and trientine dihydrochloride (Syprine); second, zinc salts to prevent the gut from absorbing copper from the diet; and third, tetrathiomolybdate which both prevents absorbing copper and binds up toxic copper in the blood making it nontoxic.

Patients who present symptomatically with mild to moderate liver failure can be effectively treated with a combination of trientine and zinc for 4-6 months and then go on maintenance therapy with zinc or trientine alone. A second choice would be penicillamine and zinc, but penicillamine has more side effects than zinc. Patients with severe liver failure may require liver transplantation. Patients who present neurologically can best be treated with tetrathiomolybdate, but it is not commercially available as yet. The second choice is zinc alone. Zinc is rather slow-acting but does not cause the drug catalyzed worsening, so common with trientine and penicillamine. Trientine and penicillamine are poor choices to treat neurologically presenting patients because of the high frequency of neurological worsening, from which many patients never recover.

Wilson's disease (WD) Emerging Drugs

To meet the current demands of the patient pool and to counter the unmet needs of the therapeutic market, drug developers are gradually shifting their attention toward Wilson's disease as a possible indication for new targeted therapies.

Several companies are working robustly on many new therapies, such as ALXN1840 (AstraZeneca), VTX-801 (Vivet Therapeutics/ Pfizer), and UX701 (Ultragenyx Pharmaceutical). The mid-stage pipeline is crowded, with several potential therapies with the imminent attention of big pharmaceutical companies for this market space.

ALXN1840 (AstraZeneca) is a potential new once-daily oral medicine in development to treat WD. It is designed to be the first targeted de-coppering therapy that selectively and tightly binds to, and removes, copper from the body's tissues and blood.

The drug is currently in Phase III trial (FOCUS), with positive high-level results from the trial. ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilization from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments. The drug has been granted Orphan Drug Designation in the US and EU for WD
The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of 10 or more years. ALXN1840, a potential new oral medicine, demonstrated approximately three times greater copper mobilization than SoC.

UX701 (Ultragenyx Pharmaceutical) is an investigational AAV type 9 gene therapy designed to deliver a stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion. The therapy has been shown in preclinical studies to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease. UX701 is currently in Phase I/II/III.

The company has also received FDA IND clearance, which allows for advancing this new gene therapy in the clinic and hopes for a new treatment for patients with WD. UX701 has the potential to directly address the underlying basis of disease by restoring the normal transport and excretion of copper.

The therapy was granted Orphan Drug Designation in the United States and European Union

Wilson's disease (WD) Market Outlook

The Wilson's disease (WD) market outlook of the report builds a detailed comprehension of the historical, current, and forecasted Wilson's disease (WD) market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.

This segment gives a thorough detail of Wilson's disease (WD) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market, and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to the publisher, Wilson's disease (WD) market in the 7MM is expected to change in the study period (2019-2032).

Key Findings

This section includes a glimpse of the Wilson's disease (WD) market in the 7MM. In the seven major markets, Wilson's disease (WD) market size was approximately USD 300 million in 2021.

The United States: Market Outlook

This section provides the total Wilson's disease (WD) market size and market size of Wilson's disease (WD) by therapies in the United States.

Wilson's disease (WD) is a rare autosomal recessive disease resulting from a loss-of-function mutation in the ATB7B copper transporter. In contrast to many neurogenetic metabolic diseases, WD can be very effectively treated in acute and chronic stages of the disease. However, early recognition is paramount because treatment delays have a much higher risk of unfavorable clinical outcomes. The disease, if left untreated, leads to copper accumulation in the body, particularly involving the brain, liver, and cornea leading to weakness, abdominal pain, jaundice, personality change, seizures, etc.

There is currently no cure for Wilson's disease. The main aim of the current treatment is to establish a negative copper balance with the life-long administration of chelating agents (D-penicillamine, trientine salts) or zinc salt. D-penicillamine is available under the brand name Cuprimine and Depen, while trientine dihydrochloride are available as Syprine and Cufence, respectively. Treatment helps to improve the manifestations of the disease over time, and its effectiveness depends on strict adherence to it throughout life. Restriction of copper-rich foods may be beneficial. Liver transplantation is the recommended therapy in cases of acute liver failure with encephalopathy or decompensated cirrhosis despite drug therapy.

The United States accounts for the largest market size of Wilson's disease (WD) compared to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

EU-5 Countries: Market Outlook

The total Wilson's disease (WD) market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are also mentioned.

Japan Market Outlook

The total Wilson's disease (WD) market size and market size by therapies of Wilson's disease (WD) in Japan are also mentioned.

Wilson's disease (WD) Drugs Uptake

This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2019-2032. The analysis covers Wilson's disease (WD) market uptake by drugs, patient uptake by therapies, and sales of each drug.

This helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Wilson's disease (WD) Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stages. It also analyses Wilson's disease (WD)'s key players involved in developing targeted therapeutics.

Major players include AstraZeneca, Vivet Therapeutics/Pfizer, and others whose key products are expected to get launched in the US market by 20XX.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging Wilson's disease (WD) therapies.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in Wilson's disease (WD) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or Wilson's disease (WD) market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

The publisher performs a Competitive and Market Intelligence analysis of Wilson's disease (WD) market using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Wilson's disease (WD), explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
• Comprehensive insight is provided into Wilson's disease (WD) epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Wilson's disease (WD) is provided, along with the assessment of new therapies that will impact the current treatment landscape
• A detailed review of the Wilson's disease (WD) market; historical and forecasted, is included in the report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies by understanding trends shaping and driving the global Wilson's disease (WD) market

Report Highlights

• In the coming years, Wilson's disease (WD) market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world, which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Wilson's disease (WD) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Wilson's disease (WD). The launch of emerging therapies will significantly impact Wilson's disease (WD) market.
• A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for Wilson's disease (WD).
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Wilson's disease (WD) Report Insights

• Patient Population
• Therapeutic Approaches
• Wilson's disease (WD) Pipeline Analysis
• Wilson's disease (WD) Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Wilson's disease (WD) Report Key Strengths

• 11-year Forecast
• 7MM Coverage
• Wilson's disease (WD) Epidemiology Segmentation
• Key Competitors
• Highly Analyzed Market
• Drugs Uptake

Wilson's disease (WD) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness

Key Questions

Market Insights:

• What was the market share (%) distribution, and how would it look in 2032?
• What would be the total market size and market size of Wilson's disease (WD) by therapies across the 7MM forecast period (2019-2032)?
• What are the key findings pertaining to the market across 7MM, and which country will have the largest Wilson's disease (WD) market Size during the forecast period (2019-2032)?
• At what CAGR is Wilson's disease (WD) market expected to grow in the 7MM forecast period (2019-2032)?
• What would be Wilson's disease (WD) market outlook across the 7MM forecast period (2019-2032)?
• What would be Wilson's disease (WD) market growth until 2032 and the resultant market size in 2032?
• How would the future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the disease risk, burdens, and unmet needs of Wilson's disease (WD)?
• What is the historical patient pool of Wilson's disease (WD) in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of Wilson's disease (WD) in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, and the UK), and Japan?
• What will be the growth opportunities in the 7MM concerning the patient population pertaining to Wilson's disease (WD)?
• Out of all 7MM countries, which country would have the highest diagnosed prevalence of Wilson's disease (WD) during the forecast period (2019-2032)?
• At what CAGR is the population expected to grow in the 7MM during the forecast period (2019-2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of Wilson's disease (WD)?
• What are the current treatment guidelines for treating Wilson's disease (WD) in the US, Europe, and Japan?
• What are Wilson's disease (WD) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, efficacy, etc.?
• How many companies are developing therapies to treat Wilson's disease (WD)?
• How many therapies are developed by each company for the treatment of Wilson's disease (WD)?
• How many emerging therapies are in the mid-stage and late stages of development to treat Wilson's disease (WD)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and licensing activities related to Wilson's disease (WD) therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies for Wilson's disease (WD) and its status?
• Which key designations have been granted for the emerging therapies for Wilson's disease (WD)?
• What are the global historical and forecasted Wilson's disease (WD) markets?

Reasons to Buy

• The report will help in developing business strategies by understanding trends shaping and driving Wilson's disease (WD) market
• To understand the future market competition in Wilson's disease (WD) market and an Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Wilson's disease (WD) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identifying upcoming solid players in the market will help devise strategies that will help get ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for Wilson's disease (WD) market.
• To understand the future market competition in Wilson's disease (WD) market.