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, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).
The Homocystinuria market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current 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 (HCU) is an inherited rare amino acid metabolism disorder in which the body is unable to process amino acid methionine properly, leading to an abnormal accumulation of homocysteine and its metabolites in blood and urine. Normally, these metabolites are not found in appreciable quantities in blood or urine. The condition may result from a deficiency of any of several enzymes involved in the conversion of methionine to cysteine. The genes associated with HCU are CBS, MTHFR, MTR, MTRR, and MMADHC. Mutations in the CBS gene cause the most common form of HCU, known as Classical Homocystinuria.
According to the NORD, homocystinuria due to CBS deficiency is a rare disorder that affects males and females in equal numbers. Population studies suggest that the worldwide frequency of homocystinuria in the general population is one in 344,000.
Cystadane (Betaine anhydrous for oral solution) is the first agent approved by the FDA to treat Homocystinuria involving CBS, MTHFR, or cbl defects. It is considered adjunctive therapy in patients with abnormal levels of homocysteine.
Homocystinuria Market Key Facts
In the year 2017, the total prevalent cases of Homocystinuria were 41,600+ cases in the 7MM.
According to DelveInsight’s analysis, males and females are equally affected by homocystinuria.
As per the DelveInsight estimates, the US, in 2017, accounted for 32,500+ prevalent cases (the highest number).
In the United Kingdom, the total gender-specific prevalent cases of Homocystinuria were 158 cases for males and 163 for females in 2017.
According to the research study of Linnebank et al. (2001), a higher prevalence rate was observed in Germany, which is around 1 in 17,800.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Homocystinuria market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Homocystinuria market trend for each marketed 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 epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and and views of key opinion leaders.
Homocystinuria Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Homocystinuria market or expected to get 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 drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on 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 companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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The key companies in the Homocystinuria (HCU) market include:
Recordati Rare Diseases Inc.
And many others.
Homocystinuria Therapies covered in the report include:
OT-58 – OT-58 is being developed as the lead program of Orphan technologies for patients suffering from the rare disease classical HCU. OT-58 is designed to help patients reduce their homocysteine levels, improve patient health and restore a normal lifestyle.
ACN00177 – ACN00177 is the clinical candidate being developed by Aeglea BioTherapeutics. ACN00177 is as an engineered human enzyme designed to treat HCU. It is anticipated that the enzyme therapy with ACN00177 may degrade homocysteine and the oxidized form of homocysteine in the blood.
And many others.
Further Emerging Drugs & Key Companies are Covered in the Report. Download the Sample PDF to Learn More:
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 Epidemiology and Patient Population (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 Products
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 Access and Reimbursement Overview
17. KOL Views on the Homocystinuria Market
18. Homocystinuria Market Drivers
19. Homocystinuria Market Barriers
21. DelveInsight Capabilities
*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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