DelveInsight’s “Homocystinuria Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Homocystinuria market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Homocystinuria drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Homocystinuria treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Homocystinuria: An Overview
Homocystinuria(HCY) is a rare, autosomal recessively hereditary disorder that caters to a faulty methionine metabolism, resulting in an unusual accumulation of homocysteine and its metabolites such as homocysteine, homocysteine-cysteine complexes, etc. The reserves of homocysteine can be found in the blood and urine in large quantities. The common forms of HCY can be identified when a person experiences myopia, osteoporosis, megaloblastic anemia, fits, immobility, and clotting of blood. It is caused due to certain mutations occurring in the genetic makeup of a human comprising of mutations in the CBS, MTHFR, MTR, MTRR, AND MMADHC genes. The CBS gene directs the enzyme, cystathionine beta-synthase which effectively converts homocysteine to cystathionine. Mutations in the CBS gene result in the formation of abnormal reserves of homocysteine and other toxic components in the blood and urine. There are three main forms of Homocystinuria: Homocystinuria I, Homocystinuria II, and Homocystinuria III.
The symptoms of Homocystinuria include trouble in learning, lens dislocation in eyes, blood clotting, brittle bones (osteoporosis), developmental issues, scoliosis, chest protrusion, irregular widened metaphysis, Abnormal size and shape of the epiphysis, Pes cavus, High-arched palate, Metaphyseal spicules, etc.
The treatment of Homocystinuria consists of Pyridoxine therapy (vitamin B6), Betaine therapy (Cystadane®), and supplements with folate(vitamin B9) and cobalamin (vitamin B12)
Homocystinuria Market Key Facts
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As per the study titled “Epidemiological study of the metabolic diseases with homocystinuria in Spain”, 2012, conducted by García-Jiménez et al. Homocystinuria incidence was found to be much higher than the earlier reported incidences (1 in 20,000 newly born).
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As per a study, the incidence rates in Ireland, Austria, Great Britain, and New England were 1 in 50,000 and the incidence rate in Japan was estimated to be around 1 in 10,00,000.
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According to the National Organization for Rare Disorders data, the prevalence in Germany has been found to be 1 in 17,500. Studies in different European countries have also given the statistical data entailing the homocystinuria happening due to the faulty CBS gene is as high as 1 in 6,400 to 1 in 20,000.
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As per the study conducted by Taylor et al., “Ophthalmic abnormalities in homocystinuria: the value of screening” for homocystinuria(HCY) the prevalence accounts for about 1 in 52,544 births in Ireland, 1 in 96,000 in the UK (Manchester) and about 1 in 4,06,000 in the United States. The prevalence found in the United States was correlated to the patients with Celtic ancestry.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Homocystinuria pipeline therapies. It also thoroughly assesses the Homocystinuria market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Homocystinuria drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Homocystinuria Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Homocystinuria epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Homocystinuria epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Homocystinuria Epidemiology, Segmented as –
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Prevalent cases of Homocystinuria in 7MM (2019–2032)
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Age-specific Prevalent Cases of Homocystinuria in 7MM (2019–2032)
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Diagnosed & Treatable Cases of Homocystinuria in 7MM (2019–2032)
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Gender-specific Prevalent Cases of Homocystinuria in 7MM (2019–2032)
Homocystinuria Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Homocystinuria market or expected to be launched during the study period. The analysis covers the Homocystinuria market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Homocystinuria drugs based on their sale and market share.
The report also covers the Homocystinuria pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Homocystinuria companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Homocystinuria Therapeutics Analysis
The treatment protocols for Homocystinuria depend on the age at which diagnosis of Homocystinuria takes place. The diagnosis of the disorder in an early infant would automatically shift the treatment in order to prevent the ocular, skeletal, and any kind of intravascular thromboembolic complications, developmental disabilities related to learning, and normal intelligence.
However, in case of late-stage diagnosis, the treatment follows a protocol to reduce the already escalating symptoms comprising life-threatening symptoms like thromboembolic events and to reduce further damage. The main forms of treatment for Homocystinuria include Pyridoxine, Vitamin B12 (Cobalamin) and Folates, Methionine-restricted dietary management, Betaine (Cystadane.
The current pipeline for Homocystinuria does not hold many significant products, it has very few products that are being developed by certain key players. One of the potential emerging therapies for Homocystinuria is OT-58, under development by Orphan Technologies Ltd.
Homocystinuria Companies Actively Working in the Therapeutics Market Include
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OT-58: Orphan Technologies Ltd.
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ACN 00177: Aeglea Biotherapeutics
And Many Others
Emerging and Marketed Homocystinuria Therapies Covered in the Report Include:
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Orphan Technologies Ltd.
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Aeglea Biotherapeutics
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Homocystinuria Competitive Intelligence Analysis
4. Homocystinuria Market Overview at a Glance
5. Homocystinuria Disease Background and Overview
6. Homocystinuria Patient Journey
7. Homocystinuria Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Homocystinuria Treatment Algorithm, Current Treatment, and Medical Practices
9. Homocystinuria Unmet Needs
10. Key Endpoints of Homocystinuria Treatment
11. Homocystinuria Marketed Therapies
12. Homocystinuria Emerging Drugs and Latest Therapeutic Advances
13. Homocystinuria Seven Major Market Analysis
14. Attribute Analysis
15. Homocystinuria Market Outlook (In US, EU5, and Japan)
16. Homocystinuria Companies Active in the Market
17. Homocystinuria Access and Reimbursement Overview
18. KOL Views on the Homocystinuria Market
19. Homocystinuria Market Drivers
20. Homocystinuria Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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