Menkes Disease - Market Insights, Epidemiology and Market Forecast - 2034

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.

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 Menkes Disease market 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.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Menkes Disease Disease Understanding and Treatment Algorithm

Menkes Disease Overview

Menkes disease is a lethal infantile neurodegenerative disorder with X-linked inheritance. It is an inherited X-linked recessive disorder caused by mutations in the ATP7A gene that is responsible for the transport of copper throughout the body. The quality of life (QoL) of Menkes patients deteriorates rapidly through growing severity, symptoms, and outcomes. The newborn may experience seizures, low muscle tone, twisted blood vessels, and many other defects in organs. Also, the mortality rate of patients with Menkes is very high. Seeing their newborn child in such a deadly situation is very painful for parents and families as well. Overall financial and emotional support to families is highly needed. Counseling the family about the ongoing situation and the possibility of occurrence of disease in the family should be done.

Menkes Disease Diagnosis

The diagnosis is the most formidable challenge in Menkes. Test such as biochemical evaluation is a useful tool to estimate the serum copper and ceruloplasmin levels in the patient, which are generally found lower in affected cases. But the levels of copper at birth may overlap with normal, resulting in misdiagnosis. Other most available diagnosis techniques are Neuroimaging, EEG, and others, which are performed after suspected symptoms appear. Diagnosis techniques with high accuracy and precision, such as gene-based tests, are the need of the hour.

Menkes Disease Treatment

The patients eligible for pharmacological treatment have no approved agents to treat Menkes disease in the present scenario. Few drugs are being explored in the emerging pipeline. However, looking over the abandoned space in the treatment paradigm and increasing prevalence, the emerging pipeline does not have sufficient candidates. Many accurate and targeted treatments are required to fill this unmet gap.

Menkes Disease Epidemiology

As the market is derived using the patient-based model, the Menkes Disease epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by, Diagnosed Prevalent Cases of Menkes Disease, in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• 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

Disorders of copper homeostasis are currently recognized across the life span. Their recognition and links to human disease have spanned several decades, beginning with the recognition of a degenerative disorder in the offspring of sheep grazing in copper-deficient pastures, through to the description of infants suffering from a progressive neurodegenerative disorder characterized by epileptic seizures, developmental regression, failure to thrive, and an unusual hair quality (giving the condition its distinctive label of kinky hair disease).

Menkes disease development is unpredictable and rapid and often requires medical assistance. Unfortunately, the prognosis of these patients is poor; unless there is access to copper histidine or other copper replacement therapy, survival generally does not exceed 3.5 years. Currently the management of Menkes disease is based on standard of care including off-label copper supplements (copper histidine or copper chloride), antiepileptic drugs, L-threo-dihydroxyphenylserine, and others.

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 Development 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.

Pipeline Development Activities

The report covers detailed information on collaborations, acquisitions and mergers, licensing, and patent details for Menkes Disease emerging therapies.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on Menkes Disease evolving treatment landscape, patient reliance on conventional therapies, patient’s therapy switching acceptability, and drug uptake along with challenges related to accessibility, include The Ohio State University College of Medicine, Columbus, Ohio, United States; University of Iowa, Iowa City, Iowa, the US; University Clinic of Marburg, Marburg, Germany; Department of Immunology, Institut Pasteur, France; Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan, and others.

The analysts connected with 50+ KOLs to gather insights, however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or Menkes Disease market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis, and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis is done to analyze multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

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

Menkes Disease Report Insights

• Patient Population
• Therapeutic Approaches
• Menkes Disease Pipeline Analysis
• Menkes Disease Market Size and Trends
• Existing and future Market Opportunity

Menkes Disease Report Key Strengths

• 11 Years Forecast
• 7MM Coverage
• Menkes Disease Epidemiology Segmentation
• Key Cross Competition
• Attribute analysis
• Drugs Uptake and Key Market Forecast Assumptions

Menkes Disease Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions

Market Insights:

• What was the Menkes Disease total market size, the market size by therapies, and market share (%) distribution in 2020, and how it would all look in 2034? What are the contributing factors for this growth?
• What are the unmet needs are associated with the current treatment market of Menkes Disease?
• How is CUTX-101 (Copper Histidinate)going to contribute to the market of Menkes Disease after approval?
• Which drug is going to be the largest contributor in 2034?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the disease risk, burden, and unmet needs of Menkes Disease? What will be the growth opportunities across the 7MM concerning the patient population of Menkes Disease?
• What is the historical and forecasted Menkes Disease patient pool in the United States, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan?
• Why do only limited patients appear for diagnosis? Why is the current year diagnosis rate not high?
• What factors are affecting the diagnosis and treatment of the indication?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of Menkes Disease? What are the current treatment guidelines for the treatment of Menkes Disease in the US and Europe?
• How many companies are developing therapies for the treatment of Menkes Disease?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Menkes Disease?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the key designations that have been granted for the emerging therapies for Menkes Disease?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies? Focus on reimbursement policies.
• What are the 7MM historical and forecasted market of Menkes Disease?

Reasons to buy

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Menkes Disease Market
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the Conjoint analysis section to provide visibility around leading classes
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs
• To understand the perspective of Key Opinion Leaders’ around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in future
• Detailed insights on the unmet need of the existing market so that the upcoming players can strengthen their development and launch strategy