Wilson’s disease occurs equally in both male and female population, but research studies have cited differences in the sex specific phenotypes. Hepatic symptoms occurs 55%-60% more in the female population. Frank psychosis is more prevalent in the male population which is often mistaken as a bipolar disorder or schizophrenia. In the near future gene therapy can be used to transfer ATPB7 gene if sufficient transgene of ATPB7 is expressed in the liver cells for longer duration of time. It is very important for the accurate diagnosis of Wilson’s disease or could lead to fatal consequences if not done appropriately.
The early diagnosis of Wilson’s disease is primarily based on the multiple indications such as hepatic, neuropsychiatric, ophthalmic and others. The chelators are the first line of therapy which includes Penicillamine and trientine. Zinc acetate is considered as the only mineral prescribed for the patients that are resistant to chelators therapy. Data mining and market estimation is covered in the scope of the report to understand the opportunity for novel drug discovery in the treatment of Wilson’s disease.
Market estimation and data mining is helpful in understanding the current treatment regimen and regulatory norms prevalent in different geographical regions.
Attractive investment proposition gives genuine understanding of the disease prevalence in different countries through data mining and the approach adopted to consider the treatment regimen for Wilson’s disease. Product portfolio gives an idea about the different dietary supplements enriched in zinc primarily used for the management of Wilson’s disease. Recent news coverage helps us to understand the strategic partnership taking place in the healthcare segment to lunch novel drug discovery for the treatment of Wilson’s disease. The manufacturers that are active in the treatment of Wilson’s disease are Kadmon Holdings, Inc., Merck & Co., Noble Pharma Co., Ltd., Teva Pharmaceutical Industries Limited, Tsumura & Co., Valeant Pharmaceuticals International, Inc., VHB Life Sciences, Inc. and Wilson Therapeutics AB.
Wilson’s disease is a rare hereditary disorder which occurs due to the accumulation of copper in different body organs such as liver, brain, eyes and other vital organs. The disease takes place due to the mutation in ATP7B gene. The prevalence rate of Wilson’s disease is 1 in 30,000 persons, usually diagnosed in young and adult age group. Hepatic indications are first diagnosed in the disease manifestation of Wilson’s disease on account of reduced biliary excretion of copper results in excessive accumulation of copper in liver. The early symptoms manifested in liver indications are tiredness, hepatitis with elevated levels of gamma-glutamyl transpeptidase, alanine aminotransferase and aspartate aminotransferase. The neuropsychiatric symptoms are manifested in the later stages of the disease manifestation with the basal ganglia hampering the coordinate movement. The major symptoms are Parkinsonism, tremors, ataxia, dystonia and seizures. In approximately 90 % of the patients exhibiting neuropsychiatric symptoms shows the development of Kayser-Fleishcer rings (deposition of copper) in the cornea region of the eyes. The manufacturers that are active in the treatment of Wilson’s disease are Kadmon Holdings, Inc., Merck & Co., Noble Pharma Co., Ltd., Teva Pharmaceutical Industries Limited, Tsumura & Co., Valeant Pharmaceuticals International, Inc., VHB Life Sciences, Inc. and Wilson Therapeutics AB.
The primary diagnosis of Wilson’s disease is complicated as the symptoms are similar to other diseases such as hepatitis C, seizures, jaundice, heavy metal poisoning and cerebral palsy. Even though Wilson’s disease is a genetic hereditary disease but a person is not likely to acquire the ATPB7 mutation on receiving only one abnormal gene from either of the parents. The disease management is entirely focused on compensating for the abnormal copper metabolism. As if now the chelators are the first line of treatment for achieving symptomatic relief in patients suffering with Wilson’s disease. Chelators deplete the excessive copper levels in the body by expediting their excretion through urine, but being nonspecific to copper they bind and excrete other micronutrients form the body. The adverse effects related to chelators are severe such as bone marrow suppression and kidney complications thereby hampering its market growth. Zinc acetate is the only mineral recommended as an alternative therapy to chelators. It works by reducing the dietary uptake of copper in the gastrointestinal tract and excretes it through feces. Its effect are extremely slow therefore used for maintenance therapy only.
According to the U.S. Department of Health and Human Services in 2019 countrywide survey the prevalence of Wilson’s disease in the United States is 1 in 55,000 births with the Wilson’s disease allele frequency rate of 0.00428. North America is the most significant market in the Wilson’s disease treatment drugs market on account of high Wilson’s disease heterozygote frequency rate of 0.855% in the Caucasian population. The autosomal recessive rate of Wilson’s disease is approximately 25% in Europe with patients being resistant to chelators therapy and E.U. approving only zinc acetate for Wilson’s disease management in Europe. Being an orphan disease the European Medical Agency is proactively investing in the novel drug discovery for Wilson’s disease. The neuropsychiatric indications related to Wilson’s disease are on a rise in Asia Pacific. The market is heavily dependent on treatment drugs imported from U.S. and Europe which creates a huge opportunity for the existing generic market in Asia Pacific.
Historical & Forecast Period
This study report represents analysis of each segment from 2022 to 2032 considering 2023 as the base year. Compounded Annual Growth Rate (CAGR) for each of the respective segments estimated for the forecast period of 2024 to 2032.
The current report comprises of quantitative market estimations for each micro market for every geographical region and qualitative market analysis such as micro and macro environment analysis, market trends, competitive intelligence, segment analysis, porters five force model, top winning strategies, top investment markets, emerging trends and technological analysis, case studies, strategic conclusions and recommendations and other key market insights.
Research Methodology
The complete research study was conducted in three phases, namely: secondary research, primary research, and expert panel review. key data point that enables the estimation of Wilson’s Disease Drugs market are as follows:
Market forecast was performed through proprietary software that analyzes various qualitative and quantitative factors. Growth rate and CAGR were estimated through intensive secondary and primary research. Data triangulation across various data points provides accuracy across various analyzed market segments in the report. Application of both top down and bottom-up approach for validation of market estimation assures logical, methodical and mathematical consistency of the quantitative data.
| ATTRIBUTE | DETAILS |
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| Research Period | 2022-2032 |
| Base Year | 2023 |
| Forecast Period | 2024-2032 |
| Historical Year | 2022 |
| Unit | USD Million |
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Region Segment (2022-2032; US$ Million)
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Key questions answered in this report
