The emerging pipeline for the treatment of Menkes Disease is not robust. Very few candidates are being investigated by pharmaceutical companies in this disease area. Key players, such as Sentynl Therapeutics along with Cyprium Therapeutics, and some others, are developing drugs for Menkes Disease.
DelveInsight’s “Menkes Disease Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Menkes Disease 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 Menkes Disease 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.
Menkes Disease Overview
Menkes disease, also referred to as Menkes syndrome, stems from a mutation within the ATP7A gene, influencing the body’s copper transportation and maintenance. This disorder predominantly affects male infants, manifesting in coarse, kinky hair, hence its colloquial moniker, “kinky hair disease,” alongside growth and developmental issues.
Pathogenic variants in the ATP7A gene, located at Xq21.1, encode a membrane-bound protein responsible for copper transportation (Cu2±transporting ATPase-alpha polypeptide). Approximately 300 distinct variants within this gene have been identified thus far, yet no clear correlation exists between these variants and the clinical progression of the disease.
Symptoms of Menkes disease typically emerge in infants around two to three months post-birth. However, low copper levels at birth don’t invariably indicate Menkes disease, as many otherwise healthy infants may experience temporarily low copper levels.
Menkes Disease Market Key Facts
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National Organization for Rare Disorders (NORD) (n.d.) has defined Menkes syndrome as an inherited X-linked recessive disorder that affects many systems in the body. The possibility of occurrence of Menkes disease is about 1 in 35,000 live male births each year. Most diagnosed infants are male; however, the disease may still occur in females, related to unusual genetic circumstances.
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According to Oprhanet (n.d.), the prevalence of Menkes syndrome at birth is estimated at 1/300,000 and 1/360,000 in Europe and Japan, respectively. The disorder is X-linked and thus primarily affects males.
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As per the study by Kaler et al. (2020) stated that assuming Harvey-Weinberg equilibrium, the allelic frequency of deleterious ATP7A variants in a genomic database from a large diverse population predicts a birth prevalence of Menkes disease or ATP7Arelated disorders as high as 1 in 8664 live male births.
Menkes Disease Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Menkes Disease 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 Menkes Disease 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.
Menkes Disease 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 views of key opinion leaders.
The Report Covers the Menkes Disease Epidemiology, Segmented by –
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Total Prevalent Cases of Menkes Disease in the 7MM [2019–2032]
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Diagnosed and Treatable Cases of Menkes Disease in the 7MM [2019–2032]
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Gender-Specific Prevalent Cases of Menkes Disease in the 7MM [2019–2032]
Menkes Disease Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the rate of uptake of the potential drugs recently launched in the Menkes Disease market or expected to be launched during the study period. The analysis covers the Menkes Disease 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 Menkes Disease 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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Menkes Disease Therapeutics Analysis
The Leading Companies in the Menkes Disease Therapeutics Market Include:
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Sentynl Therapeutics
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Cyprium Therapeutics
And Many Others
Menkes Disease Drugs Covered in the Report Include:
CUTX-101 (Copper Histidinate) – Cyprium Therapeutics, in collaboration with Sentynl Therapeutics, is developing CUTX-101, a copper histidine injection to treat Menkes disease. CUTX-101 has been granted Orphan Drug designation and Rare Pediatric Disease designation by the FDA for the treatment of Menkes disease and Fast Track Designation for classic Menkes disease in patients who have not demonstrated significant clinical progression. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products also granted Orphan Drug Designation for CUTX-101.
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @ https://www.delveinsight.com/sample-request/menkes-disease-market
Table of Contents
1. Key Insights
2. Executive Summary
3. Menkes Disease Competitive Intelligence Analysis
4. Menkes Disease Market Overview at a Glance
5. Menkes Disease Disease Background and Overview
6. Menkes Disease Patient Journey
7. Menkes Disease Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Menkes Disease Treatment Algorithm, Current Treatment, and Medical Practices
9. Menkes Disease Unmet Needs
10. Key Endpoints of Menkes Disease Treatment
11. Menkes Disease Marketed Products
12. Menkes Disease Emerging Drugs and Latest Therapeutic Advances
13. Menkes Disease Seven Major Market Analysis
14. Attribute Analysis
15. Menkes Disease Market Outlook (In US, EU5, and Japan)
16. Menkes Disease Access and Reimbursement Overview
17. KOL Views on the Menkes Disease Market
18. Menkes Disease Market Drivers
19. Menkes Disease Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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