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 report covers emerging Menkes Disease drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Menkes Disease 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: An Overview
Menkes disease (MD), also known as Menkes syndrome, is a disorder caused by a mutation of the ATP7A gene. This ATP7A gene affects how the body transports copper and maintains copper levels. Menkes disease occurs mostly in male infants and is characterized by coarse, kinky hair (leading to its nickname of “kinky hair disease”), as well as failure to grow and thrive.
It is caused by pathogenic variants in ATP7A (Xq21.1) encoding a membrane-bound copper-transport protein (Cu2+-transporting ATPase-alpha polypeptide). To date, about 300 different variants in this gene have been reported. There is no obvious correlation between the variants and the clinical course.
Menkes disease may not be obvious in infants until two to three months after birth when signs typically begin to appear. Low copper levels at birth do not necessarily indicate Menkes disease, since many otherwise healthy infants may be born with temporarily low copper levels.
Menkes disease is typically diagnosed based on the clinical features, medical examination, and genetic testing for alterations in the ATP7A gene. Other types of tests that may be helpful to include analysis of catecholamines (chemicals that are sensitive to copper) and copper levels in the blood
Treatment with daily copper injections may improve the outcome in Menkes disease if commenced within days after birth; however, newborn screening for this disorder is not available, and early detection is difficult because clinical abnormalities in affected newborns are absent or subtle.
Menkes Disease Market Key Facts
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Among the EU-5 countries, Germany and France had the highest market size for Menkes Disease in 2021.
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The United States had 79 diagnosed prevalent cases of Menke disease in 2021.
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In 2021, the EU-5 accounted for 57 diagnosed prevalent cases of Menkes Disease.
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Among the European countries, France had the highest diagnosed prevalent population of Menkes Disease with 15 cases, followed by Germany and UK with 14 cases in each country, in 2021. On the other hand, Spain had the lowest diagnosed prevalent population.
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In 2021, Japan had 19 diagnosed cases of Menkes Disease.
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The expected launch of potential therapies may increase market size in the coming years, assisted by an increase in the diagnosed prevalent population of Menkes Disease.
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Upcoming therapies such as CUTX-101 have the potential to create a significant positive shift in the Menkes Disease market size.
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The United States accounts for the largest market size of Menkes Disease, in comparison to other 7MM.
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 Menkes Disease pipeline therapies. It also thoroughly assesses the Menkes Disease 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 Menkes Disease 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 Menkes Disease epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Menkes Disease 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 Menkes Disease Epidemiology, Segmented as –
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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 uptake rate of 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 market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Menkes Disease drugs based on their sale and 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 Menkes Disease 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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Menkes Disease Therapeutics Analysis
There is no prevention or cure for Menke’s disease. Patients with a family history of the disorder, are recommended to talk to a genetic counselor when deciding whether to have children.
Early (ideally within 28 days of age of birth, corrected for prematurity) treatment of Menkes disease is essential. Since newborn screening for this disorder is not available, and early detection is infrequent because the clinical signs of Menkes disease are subtle in the beginning, early treatment is very challenging.
Menkes Disease Companies Actively Working in the Therapeutics Market Include
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Cyprium Therapeutics
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Sentynl Therapeutics
And Many Others
Emerging and Marketed Menkes Disease Therapies Covered in the Report Include:
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CUTX-101: Cyprium Therapeutics and Sentynl Therapeutics
CUTX-101 (Copper Histidinate) is in development to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate that is intended to improve tolerability due to its physiological pH, and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease.
In the Phase I/II clinical study conducted at the National Institute of Child Health and Human Development (NICHD), early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase III study of CUTX-101 is ongoing at NICHD.
CUTX-101 has been granted orphan drug designation by the FDA. FDA granted Fast Track Designation and Rare Pediatric Disease Designation to CUTX-101. In July 2020, European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the Orphan Medicinal Product Designation for CUTX-101. If approved, it will become an FDA-approved treatment for Menkes disease.
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Table of Content (TOC)
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 Patient Population and Epidemiology Trends (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 Therapies
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 Companies Active in the Market
17. Menkes Disease Access and Reimbursement Overview
18. KOL Views on the Menkes Disease Market
19. Menkes Disease Market Drivers
20. Menkes Disease 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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