PUBLISHER: DelveInsight | PRODUCT CODE: 1108656
PUBLISHER: DelveInsight | PRODUCT CODE: 1108656
DelveInsight's, '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
Study Period: 2019-2032
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.
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.
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.
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.
Products detail in the report…
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-naive 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
Products detail in the report…
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 DelveInsight, 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.
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.
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.
The total Wilson's disease (WD) market size and market size by therapies of Wilson's disease (WD) in Japan are also mentioned.
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
We perform 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.
Key Questions
Market Insights:
Epidemiology Insights:
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies: