Dublin, Dec. 11, 2024 (GLOBE NEWSWIRE) -- The "Menkes Disease - Market Insights, Epidemiology and Forecast to 2034" report has been added to ResearchAndMarkets.com's offering.
This report delivers an in-depth understanding of the Menkes Disease, historical and forecasted epidemiology as well as the Menkes Disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Menkes Disease market size from 2020 to 2034. The report also covers current Menkes Disease treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Key Highlights:
- Menkes disease diagnosed prevalence is expected to rise, driven by advancements in genetic testing, increased awareness, improved survival rates, genetic factors, expanded screening, and environmental influences.
- In 2023, the US accounted for the maximum diagnosed patient share of Menkes disease in the 7MM, i.e., 54%, followed by Japan accounting for around 11% of the total 7MM cases. Among the European countries higher number of cases were in France accounting for about 9% of the total 7MM cases.
- In 2023, the market size of Menkes Disease was highest in the US among the 7MM accounting for approximately USD 7 million that is further expected to increase by 2034.
- The overall market was covered by Standard of Care, making up the total market of around USD 7.9 million in 2023, in the 7MM.
- Although the prevalence of Menkes Disease has been steadily rising, with approximately 177 diagnosed cases reported in the 7MM in 2023, there remains a significant gap in approved therapies tailored specifically for its treatment.
Menkes Disease Epidemiology
- As per our assessment, in 2023, nearly 177 diagnosed prevalent cases of Menkes Disease were estimated in the 7MM. These cases are anticipated to increase by 2034. The US accounted for the highest cases.
- Among the European countries, France (26%) had the highest diagnosed prevalent population of Menkes Disease, followed by Germany and the UK (24%) in 2023. On the other hand, Spain (11%) had the least diagnosed prevalent population of Menkes Disease in the same year.
- In 2023, Japan accounted for around 19 diagnosed prevalent cases of Menkes disease.
Menkes Disease Drug Chapters
The drug chapter segment of the Menkes Disease report encloses a detailed analysis of Menkes Disease marketed drugs and late-stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Menkes Disease clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
Emerging Drugs
CUTX-101: Cyprium Therapeutics/Sentynl Therapeutics
CUTX-101 is under investigation for the treatment of Menkes disease and related disorders. It has not yet been established as safe and effective for any such treatment by the US FDA or any other regulatory authorities. However, it is available through an Expanded Access Protocol to obtain further safety and efficacy data that may be used to support the approval of the product and subsequent wider accessibility to Menkes disease patients. The drug received designations from the FDA like Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations.
Additionally, the European Medicines Agency granted Orphan Drug Designation for CUTX-101. In December 2023, Cyprium completed the transfer of its proprietary rights and assigned its FDA documents pertaining to CUTX-101 Copper Histidinate product candidate for the treatment of Menkes disease, to Sentynl Therapeutics, Inc. A rolling NDA submission to the FDA for CUTX-101 is underway and expected to be completed by Sentynl in 2024.
Drug Class Insights
The current treatment for Menkes disease involves copper supplementation, usually administered through injections or oral medication. This aims to address the copper deficiency characteristic of the condition. Early intervention is critical for optimal outcomes, as treatment effectiveness diminishes with disease progression. While copper therapy can alleviate some symptoms and improve quality of life, it does not fully reverse the neurological damage already incurred. Supportive therapies, such as physical and occupational therapy, may also be employed to manage symptoms and enhance overall well-being.
Menkes Disease Market Outlook
The advancement in therapeutic approaches open the market for new emerging therapies. New therapeutic approaches for Menkes disease are under development CUTX-101, and others.
Until now, CUTX-101 seems to be the most promising candidate in the clinical pipeline of Menkes disease. The market is expected to react positively to CUTX-101, aiding it in solidifying its position as a leading therapy for this indication, as no other therapy has been able to pave its way to approval yet. We believe the expected approval of emerging therapy and awareness regarding early screening of the disease will allow the market to rise at a significant rate.
- The total market size of Menkes disease in the 7MM was approximately USD 7.9 million in 2023 and is projected to increase during the forecast period (2024-2034).
- The market size in the 7MM will increase at a constant CAGR due to increasing awareness of the disease, better diagnosis, and the launch of the emerging target therapy for Menkes disease.
- Among EU4 and the UK, France accounted for the maximum market share of 27% in 2023 while Spain occupied the bottom of the ladder with aa share of 12% in 2023.
- The 7MM treatment market lacks approved targeted treatment for Menkes Disease, but with the expected launch of CUTX-101 by 2025 the market is expected to experience positive growth.
Menkes Disease Drugs Uptake
This section focuses on the rate of uptake of the potential drugs expected to get launched in the market during the study period 2020-2034. For example, for CUTX-101 (Copper Histidinate), we expect the drug uptake to be medium-fast with a probability-adjusted peak share of around 80%, and years to the peak is expected to be 5 years from the year of launch in the US.
Menkes Disease Pipeline Activities
The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I stage. It also analyzes key players involved in developing targeted therapeutics.
Menkes Disease Development Activities
The report covers detailed information on collaborations, acquisitions and mergers, licensing, and patent details for Menkes Disease emerging therapies.
Market Access and Reimbursement
Reimbursement is a crucial point for any drug after its approval. Many drugs or therapies are not properly recognized by the reimbursement body and may fail to get reimbursed or their reimbursement process gets delayed.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
Scope of the Report
- The report covers a segment of key events, an executive summary, descriptive overview of Menkes Disease, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies
- Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression along with treatment guidelines
- Additionally, an all-inclusive account of both the current and emerging therapies along with the elaborative profiles of late-stage and prominent therapies will have an impact on the current treatment landscape
- A detailed review of the Menkes Disease market; historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach
- The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preference that help in shaping and driving the 7MM Menkes Disease market
List of Chapters
1. Key Insights
2. Report Introduction
3. Menkes Disease Market Overview at a Glance
4. Epidemiology and Market Forecast Methodology of Menkes Disease
5. Executive Summary of Menkes Disease
6. Key Events
7. Disease Background and Overview
8. Epidemiology and Patient Population
9. Patient Journey
10. Emerging Drugs
11. Menkes Disease: Seven Major Market Analysis
12. KOL Views
13. SWOT Analysis
14. Unmet Needs
15. Market Access and Reimbursement
16. Appendix
For more information about this report visit https://www.researchandmarkets.com/r/w3e9ku
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