Charcot Marie Tooth Disease Market
DelveInsight’s ‘Charcot–Marie–Tooth Disease -Market Insights, Epidemiology, and Market Forecast–2030’ report deliver an in-depth understanding of the Charcot–Marie–Tooth Disease, historical and forecasted epidemiology as well as the Charcot–Marie–Tooth Disease market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
The Charcot–Marie–Tooth Disease market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Charcot–Marie–Tooth Disease market size from 2018 to 2030. The Report also covers current Charcot–Marie–Tooth Disease treatment practice, market drivers, market barriers, SWOT analysis, reimbursement, and market access, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
- The United States
- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Study Period: 2018–2030
Charcot–Marie–Tooth Disease Disease Understanding and Treatment Algorithm
Charcot–Marie–Tooth Disease is a disease of the peripheral nerves that control muscles – unlike muscular dystrophies, which affect the muscles themselves. It is a group of genetically heterogeneous disorders that affect the motor and/or peripheral sensory nerves, resulting in muscle weakness and atrophy in addition to sensory loss. Specific gene mutations are responsible for the abnormal function of the peripheral nerves. The nerve cells in individuals with this disorder cannot send electrical signals properly because of abnormalities in the nerve axon or abnormalities in the insulation (myelin) around the axon. Distal legs followed by hands are some of the first locations which exhibit such manifestations. CMT can be inherited in an autosomal dominant, autosomal recessive, or X-linked mode of inheritance and is also known as hereditary motor and sensory neuropathy (HMSN).
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Charcot–Marie–Tooth Disease Diagnosis
The diagnosis and management of the patient with CMT are best undertaken by a multidisciplinary approach that involves a neurologist and, when appropriate, a genetic counselor, orthopedic surgeon, physiatrist, pulmonary specialist, physical therapist, occupational therapist, and/or social worker to provide the maximum care for patients with chronic disability.
The diagnosis of CMT begins with a standard medical history, family history, and neurological examination. The individuals are asked about the nature and duration of their symptoms and whether other family members have the disease. During the neurological examination, a physician looks for evidence of muscle weakness in the individual’s arms, legs, hands, and feet, decreased muscle bulk, reduced tendon reflexes, and sensory loss. In addition, the doctors look for evidence of foot deformities, such as high arches, hammertoes, inverted heels, or flat feet. Other orthopedic problems, such as mild scoliosis or hip dysplasia, may also be present. A specific sign that may be found in people with CMT1 is nerve enlargement that may be felt or even seen through the skin. These enlarged nerves, called hypertrophic nerves, are caused by abnormally thickened myelin sheaths.
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Charcot–Marie–Tooth Disease Treatment
Management of CMT is currently supportive; however, such supportive therapy can dramatically improve a patient’s quality of life. Although there is no cure for CMT, treatments can be used to manage its symptoms effectively. These treatments have allowed many people with the disease to lead active, productive lives. The main treatment for CMT involves working with an occupational or physical therapist. Optimal management is multidisciplinary, with care provided by neurologists, genetic counselors, nurses, physical and occupational therapists, physiatrists, and orthopedic surgeons. Maintaining mobility, flexibility, and muscle strength are important. Beginning a treatment program early may delay or reduce nerve degeneration and muscle weakness before it progresses to the point of disability. Physical therapy includes muscle strength training, muscle and ligament stretching, and moderate aerobic exercise. A specialized exercise program approved by the person’s physician can help build stamina, increase endurance, and maintain overall health.
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Charcot–Marie–Tooth Disease Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of Charcot–Marie–Tooth Disease, Diagnosed Prevalent Cases of Charcot–Marie–Tooth Disease, Gender-specific Diagnosed Prevalent Cases of Charcot–Marie–Tooth Disease, Type-specific Diagnosed Prevalent Cases of Charcot–Marie–Tooth Disease, and Age-specific Diagnosed Prevalent Cases of Charcot–Marie–Tooth Disease scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2018 to 2030.
- The total prevalent population of Charcot Marie Tooth disease (CMT) in seven major markets was found to be 263,835 in 2020 and is anticipated to increase in 2030.
- The estimates suggest a higher diagnosed prevalence of CMT in the United States with 95,221 diagnosed cases in 2020, which might increase in 2030.
- The estimates suggest that a higher number of males were affected by this disease.
- The most common type of CMT is CMT1. CMT1 is further subcategorized into different subtypes that include, CMT1A, CMT1B, CMT1D, etc., and among them, the most common one is CMT1A.
Country-Wise Charcot–Marie–Tooth Disease Epidemiology
The epidemiology segment also provides the Charcot–Marie–Tooth Disease epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
Charcot–Marie–Tooth Disease Drug Chapters
Charcot–Marie–Tooth Disease Emerging Drugs
PXT3003 is a novel, synergistic, low-dose fixed combination of baclofen (a muscle relaxant), naltrexone (an opioid antagonist), and sorbitol (a laxative) given twice daily as an oral solution. The product has multiple mechanisms of action: inhibition of the overexpression of the PMP22 gene associated with an improvement in myelination, preservation of the peripheral nerve axon, and additional beneficial effects on other cell types: muscle cells, neuromuscular junctions, and immune cells. Pharnext’s first-in-class Pleodrug PXT3003, developed using Pharnext’s R&D platform, Pleotherapy, and is a novel oral fixed-dose combination of baclofen, naltrexone, and sorbitol, with Orphan Drug Designation in the EU and the US.
Products detail in the report…
MD1003: MedDay Pharmaceuticals
MD1003 is an orally administered patented, high-dose Pharmaceutical-grade Biotin (hdPB), which potentially acts on two processes involved in the breakdown of the myelin sheath, activating the Krebs cycle, the main route for ATP energy production that protects against axonal degeneration and activating acetyl-CoA carboxylase, the rate-limiting enzyme in the synthesis of fatty acids required for myelin repair. MD-1003 was evaluated in Phase II clinical trial for the treatment of CMT disease. The company also concluded two Phase III studies in progressive forms of multiple sclerosis. The first pivotal study MS-SPI had met its primary endpoint. However, as reported in March 2020, the second confirmatory Phase III study “SPI2” did not meet its primary or secondary endpoints
Products detail in the report…
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Charcot–Marie–Tooth Disease Market Outlook
There is no cure and no approved therapies for the treatment of CMT. The current therapeutic market for CMT is based on supportive and symptomatic management such as occupational therapies, physiotherapy, ankle-foot orthoses (AFO), and shoe inserts. Physiotherapy is the most common aid, followed by foot care and occupational therapy. Foot surgery for correction of foot deformities remains an option of last resort in approximately ~30% of the total number of diagnosed cases.
Pharmacotherapies mainly comprise pain killers that help reduce either joint or muscle pain — caused by the stresses that CMT places on the body or neuropathic pain — caused by damage to the nerves. Neuropathic pain may be treated with tricyclic antidepressants (TCAs) or an anti-convulsant medication.
Maintaining mobility, flexibility, and muscle strength are important. Beginning a treatment program early may delay or reduce nerve degeneration and muscle weakness before it progresses to the point of disability. Physical therapy includes muscle strength training, muscle and ligament stretching, and moderate aerobic exercise. A specialized exercise program approved by the person’s physician can help build stamina, increase endurance, and maintain overall health.
Many individuals with CMT require ankle braces and other orthopedic devices to maintain everyday mobility and prevent injury. Braces can help prevent ankle sprains by providing support and stability during walking or climbing stairs. High-top shoes or boots also can give the person support for weak ankles. Thumb splints can help with hand weakness and loss of fine motor skills. Assistive devices should be used before disability sets in because the devices may prevent muscle strain and reduce muscle weakening.
Some people with CMT may decide to have orthopedic surgery to treat severe foot and joint deformities, improve the ability to walk, and lessen pain.
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- The market size of Charcot Marie Tooth disease (CMT) in seven major markets was estimated to be USD 55 million in 2020, for the study period (2018–2030).
- The United States accounts for the largest market size of CMT, in comparison to EU5, and Japan.
- The expected Launch of potential therapies may increase market size in the coming years, assisted by an increase in the diagnosed prevalent population of CMT.
- Among the EU5 countries, Germany had the highest market size with USD 4 million in 2020, while Spain and Italy had the lowest market size of CMT with USD 2 million each, in 2020.
“The current market size of Charcot Marie Tooth Disease based on the symptomatic treatment or standard of care which includes using of therapies to control the pain. Current Treatment includes medications like opioid analgesics, antidepressants & non-opioid analgesics. Some patient also goes for surgery and further take pain medications for pain. It is estimated that only one therapy is anticipated to get launched in the forecasted market, i.e., PXT3003, which is targeting CMT1A patient pool. This therapy might have the potential to create a significant positive shift in the CMT market size.”
The United States Market Outlook
This section provides the total Charcot–Marie–Tooth Disease market size and; market size by therapies in the United States.
EU-5 Market Outlook
The total Charcot–Marie–Tooth Disease market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
Japan Market Outlook
The total Charcot–Marie–Tooth Disease market size and market size by therapies in Japan are provided.
Charcot–Marie–Tooth Disease Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched in the Charcot–Marie–Tooth Disease market or expected to get launched in the market during the study period 2018–2030. The analysis covers the Charcot–Marie–Tooth Disease market uptake by drugs; patient uptake by therapies; and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Charcot–Marie–Tooth Disease Development Activities
The report provides insights into different therapeutic candidates in phase II, and phase III stages. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Charcot–Marie–Tooth Disease emerging therapies.
Reimbursement Scenario in Charcot–Marie–Tooth Disease
Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.
Competitive Intelligence Analysis
We perform competitive and market Intelligence analysis of the Charcot–Marie–Tooth Disease market by using various competitive intelligence tools that include–SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Scope of the Report
- The report covers the descriptive overview of Charcot–Marie–Tooth Disease, explaining its causes, symptoms, pathophysiology, genetic basis, and currently available therapies.
- Comprehensive insight has been provided into the Charcot–Marie–Tooth Disease epidemiology and treatment.
- Additionally, an all-inclusive account of both the current and emerging therapies for Charcot–Marie–Tooth Disease is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
- A detailed review of the Charcot–Marie–Tooth Disease market; historical and forecasted is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM Charcot–Marie–Tooth Disease market.
- The robust pipeline with novel MOA and oral ROA, increasing prevalence, effectiveness of drugs as both mono and combination therapy will positively drive the Charcot–Marie–Tooth Disease market.
- The companies and academics are working to assess challenges and seek opportunities that could influence Charcot–Marie–Tooth Disease R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
- Major players are involved in developing therapies for Charcot–Marie–Tooth Disease. The launch of emerging therapies will significantly impact the Charcot–Marie–Tooth Disease market.
- Our in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
Charcot–Marie–Tooth Disease Report Insights
- Patient Population
- Therapeutic Approaches
- Charcot–Marie–Tooth Disease Pipeline Analysis
- Charcot–Marie–Tooth Disease Market Size and Trends
- Market Opportunities
- Impact of upcoming Therapies
Charcot–Marie–Tooth Disease Report Key Strengths
- 10 Years Forecast
- 7MM Coverage
- Charcot–Marie–Tooth Disease Epidemiology Segmentation
- Key Cross Competition
- Highly Analyzed Market
- Drugs Uptake
Charcot–Marie–Tooth Disease Report Assessment
- Current Treatment Practices
- Unmet Needs
- Pipeline Product Profiles
- Market Attractiveness
- Market Drivers and Barriers
- SWOT analysis
- What was the Charcot–Marie–Tooth Disease market share (%) distribution in 2018 and how it would look like in 2030?
- What would be the Charcot–Marie–Tooth Disease total market size as well as market size by therapies across the 7MM during the forecast period (2021–2030)?
- What are the key findings pertaining to the market across the 7MM and which country will have the largest Charcot–Marie–Tooth Disease market size during the forecast period (2021–2030)?
- At what CAGR, the Charcot–Marie–Tooth Disease market is expected to grow at the 7MM level during the forecast period (2021–2030)?
- What would be the Charcot–Marie–Tooth Disease market outlook across the 7MM during the forecast period (2021–2030)?
- What would be the Charcot–Marie–Tooth Disease market growth till 2030 and what will be the resultant market size in the year 2030?
- How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
- What are the disease risk, burdens, and unmet needs of Charcot–Marie–Tooth Disease?
- What is the historical Charcot–Marie–Tooth Disease patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
- What would be the forecasted patient pool of Charcot–Marie–Tooth Disease at the 7MM level?
- What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Charcot–Marie–Tooth Disease?
- Out of the above-mentioned countries, which country would have the highest prevalent population of Charcot–Marie–Tooth Disease during the forecast period (2021–2030)?
- At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
- What are the current options for the treatment of Charcot–Marie–Tooth Disease along with the approved therapy?
- What are the current treatment guidelines for the treatment of Charcot–Marie–Tooth Disease in the US and Europe?
- What are the Charcot–Marie–Tooth Disease marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
- How many companies are developing therapies for the treatment of Charcot–Marie–Tooth Disease?
- How many emerging therapies are in the mid-stage and late stages of development for the treatment of Charcot–Marie–Tooth Disease?
- What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Charcot–Marie–Tooth Disease therapies?
- What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Charcot–Marie–Tooth Disease and their status?
- What are the key designations that have been granted for the emerging therapies for Charcot–Marie–Tooth Disease?
- What are the 7MM historical and forecasted market of Charcot–Marie–Tooth Disease?
Reasons to buy
- The report will help in developing business strategies by understanding trends shaping and driving Charcot–Marie–Tooth Disease.
- To understand the future market competition in the Charcot–Marie–Tooth Disease market and an Insightful review of the key market drivers and barriers.
- Organize sales and marketing efforts by identifying the best opportunities for Charcot–Marie–Tooth Disease in the US, Europe (Germany, Spain, Italy, France, and 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.
- Organize sales and marketing efforts by identifying the best opportunities for the Charcot–Marie–Tooth Disease market.
- To understand the future market competition in the Charcot–Marie–Tooth Disease market.