Wilson disease is a genetic disorder that prevents the removal of extra copper in the body. Without treatment, the disease can cause life-threatening organ damage due to the copper built up in the liver, eyes, brain, and other organs.
The diagnosis of Wilson disease is based on family history, physical exams, an eye exam, blood tests, and urine tests. Liver biopsy and imaging tests may also be used.
Treatment of Wilson disease is done with medication such as zinc. Patients who have Wilson disease need lifelong surgery to manage symptoms to reduce or prevent organ damage. Wilson’s disease can cause acute liver failure which may lead to the need for a liver transplant.
The global market for Wilson’s disease Treatment is primarily driven by the financial incentives and overall rewards for the successful development of drugs for the treatment and diagnosis of rare diseases and conditions.
Moreover, the increasing awareness regarding Wilson’s disease by various organization and association and its symptoms along with the growing population all over the world is expected to fuel the treatment market.
For instance, Wilson’s disease association, a volunteer organization strives to promote the latest information about Wilson’s disease and support research and clinical investigation for the illness.
Wilson disease is found worldwide, with an estimated prevalence of 1 case per 30,000 live births in most populations and approximately one person in 90 carries an abnormal copy of the ATP7B gene, which causes the diseases.
However, the high initial investments resulting in higher per patient treatment cost may hinder the market progress.
By Indication, the Global Wilson’s Disease Treatment Market is segmented into Hepatic, Neuropsychiatric, Ophthalmic, and others (heart and kidney complications).
The Hepatic indications are diagnosed in Wilson’s disease as the reduced biliary excretion of copper results in an excessive accumulation of copper in the liver.
The early symptoms are manifested in the liver as tiredness, hepatitis and suddenly develop acute liver failure. According to the European Association for the study of the liver, an estimated 6-12% of people having acute liver failure are affected by Wilson’s disease.
The neuropsychiatric indications are manifested in the later stages of the disease manifestation persistent neurological problems such as Parkinsonism, tremors, and seizures. Involuntary muscle movements, tremors, clumsy gait and speech difficulties may improve with treatment for Wilson’s disease.
In approximately 90 % of the patients exhibiting neuropsychiatric symptoms shows the development of Kayser-Fleischer rings (deposition of copper) in the cornea region of the eyes.
By Treatment, the Global Wilson’s Disease Treatment Market is segmented into medication and others. Medication share is future sub-segmented into D-Penicillamine, Trientine, Zinc, and Tetrathiomolybdate. Currently, chelators are the first line of treatment for achieving symptomatic relief. Penicillamine and trientine are two chelating agents used to treat Wilson disease. These medicines deplete the excessive copper levels from the body.
However, the side-effects associated with chelators are severe such as bone marrow suppression and kidney complications thus hampering its market growth. Zinc acetate is recommended as an alternative therapy to chelators.
However, its effect is prolonged hence used for maintenance therapy only.
North America is the dominant share followed by Europe in the global Wilson’s disease treatment market, and it is primarily attributed to factors such as rapid growth in the geriatric population in these regions.
Being an orphan disease, the European Medical Agency is proactively investing in the novel drug discovery for Wilson’s disease. Moreover, the established healthcare infrastructure and the rising disposable income and higher investment in R&D activities contribute to the growth of North America’s share.
According to a countrywide survey by U.S. Department of Health and Human Services, 2015, the prevalence of Wilson’s disease in the United States is 1 in 55,000 births with Wilson’s disease allele frequency of 0.428%.
Asia-Pacific region is expected to be the fastest growing market during the forecast period, owing to the region’s increasing population in countries such as China and India.
This makes the area highly lucrative markets for Wilson’s disease treatment. The region’s rapidly improving healthcare infrastructure, the rise in healthcare expenditure, and the increasing awareness of Wilson’s disease also boost the market.
The market is deeply dependent on imported drugs from the U.S. and Europe which creates an opportunity for the current generic market in the Asia Pacific.
Key players are adopting strategies such as mergers and acquisitions, partnerships, and regional expansion along with an increased focus on R&D to stand out as strong competitors in the market.
May 2018, Alexion Pharmaceuticals announced that its offer to acquire all its outstanding shares through a wholly-owned subsidiary of Wilson Therapeutics had been accepted Wilson Therapeutics is a biopharmaceutical company, based in Sweden, that develops novel therapies for patients with Wilson’s disease. Wilson Therapeutics’ product, WTX101, is in Phase 3 development as a treatment for Wilson diseases.
February 2018, Teva Pharmaceuticals announced the release of a generic version of Syprine (trientine hydrochloride) capsules, 250 mg, as a substitute treatment for Wilson’s disease in the United States.
December 2017, Wilson Therapeutics AB announced that the U.S. Food and Drug Administration (FDA) granted WTX101 Fast Track designation for the treatment of Wilson Disease. WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper-protein-binding agent with a unique mechanism of action, under investigation as a novel therapy for Wilson Disease.
September 2017, The French biotech, Vivet Therapeutics is developing the first gene therapy for Wilson’s disease. Seeing its potential, both the FDA and the EC have decided to grant orphan status to the treatment, which makes it easier and cheaper to get it approved if it proves to be safe and effective. The biotech is planning started the first clinical trial in late 2018.
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1.1. Research Methodology
1.2. Scope of the market
2.1. Key Trends and Developments
3.3. Supply Chain Analysis
3.5. Key Developments
4.1. By Indication
4.1.4. Others (heart and kidney complications)
4.2. By Treatment
4.3. By Distribution Channel
5.1. Geographic Overview
5.2. North America
5.2.1. The U.S.
5.3.4. Rest of Europe
5.4. South America
5.4.3. Rest of South America
5.5. Asia Pacific
5.5.5. Rest of Asia Pacific
5.6. Rest of World
6.1. Competitive Scenario
6.2. Key Strategies adopted by Manufacturers
6.3. Product Benchmarking
7.1. Tsumura & Co
7.3. VHB Life Science Inc.
7.4. Teva Pharmaceuticals
7.5. Alexion Pharmaceuticals (Wilson’s Therapeutics AB)
7.6. Noble Pharma Co., Ltd
7.7. Kadmon Holding, Inc.
7.8. Taj Pharmaceutical Limited
7.9. Vivet Therapeutics
8.2. List of Tables
8.3. Expert Panel Validation
8.5. Contact Us
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