DelveInsight’s Charcot Marie Tooth Disease Market report offers detailed information on current treatment practices, emerging drugs, Charcot Marie Tooth Disease market share of the individual therapies, current and forecasted Charcot Marie Tooth Disease market size from 2019 to 2032 segmented into 7MM (the USA, EU5 (the UK, Italy, Spain, France, and Germany), and Japan).
Key takeaways from the Charcot Marie Tooth disease Market Research Report
- Charcot Marie Tooth disease in 7MM was estimated to be USD 55 million in 2020.
- 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.
- Charcot Marie Tooth Disease market size is anticipated to witness substantial growth owing to increased awareness, increasing prevalence, the launch of emerging therapies and heightened R&D.
- Key pharmaceutical and biotech companies working to give momentum to the Charcot Marie Tooth Disease market space growth include Pharnext, MedDay Pharmaceuticals, among others.
- Charcot Marie Tooth Disease pipeline therapies expected to enter the market in the forecasted period (2021-2030) include MD1003, PXT3003 and others.
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Charcot Marie Tooth disease Overview
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).
Charcot–Marie–Tooth Disease Epidemiology Segmentation in the 7MM
- Total Charcot Marie Tooth Disease Prevalent Cases
- Total Charcot Marie Tooth Disease Cases Diagnosed
- Charcot Marie Tooth Disease Cases Gender-specific Diagnosed Charcot
- Charcot Marie Tooth Disease Cases Type-specific Diagnosed
- Charcot Marie Tooth Disease Cases Age-specific Diagnosed
Understand how Charcot Marie Tooth Disease Epidemiological Trends @ Charcot Marie Tooth Disease Epidemiology Report
Charcot Marie Tooth Disease Therapeutic Market Outlook
The current Charcot Marie Tooth Disease therapy market majorly offers supportive and symptomatic treatments such as occupational therapies, physiotherapy, ankle-foot orthoses (AFO), and shoe inserts, with physiotherapy being the most common aid followed by foot care and occupational therapy. In cases, where no option manages to bring relief, foot surgery is opted, which is approximately 30% of the total diagnosed CMT prevalent cases. Pharmacotherapies generally address neuropathic pain with the help of tricyclic antidepressants (TCAs) or anticonvulsant medication.
Charcot–Marie–Tooth Disease Treatment Market
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.
Charcot–Marie–Tooth Disease Emerging Drugs
- PXT3003: Pharnext
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.
- 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.
Learn more about available therapies @ Charcot Marie Tooth Disease Market Forecast
Charcot Marie Tooth Disease Market Dynamics
The expansion of the CMT market share is expected to get hampered attributable to difficult diagnosis. Besides, the cost also plays an important role here as the high cost associated with the diagnosis dejects patients to undergo the same. There exists a huge unmet need in terms of effective, safe and curative therapies. Additionally, a better stratification of the patients during the clinical studies, such as layering based on the molecular effects of mutations, has posed a significant challenge during the management of the study coupled with the implementation of a combined treatment protocol.
Charcot Marie Tooth Disease Pipeline Therapies and Companies
- PXT3003: Pharnext
- MD1003: MedDay Pharmaceuticals
Discover more about the future market share of the report @ Charcot Marie Tooth Disease Treatment Market Landscape
Scope of the Charcot Marie Tooth Disease Market Report
- Coverage– 7MM
- Study Period– 2019-2032
- Charcot Marie Tooth Disease Companies– Pharnext Company (NYSE: PHARN), MedDay Pharmaceuticals, and others.
- Charcot Marie Tooth Disease Pipeline Therapies- MD1003, PXT3003, and others.
- Charcot Marie Tooth Disease Market Dynamics: Charcot Marie Tooth Disease Market Drivers and Barriers
Table of Content
- Key Insights
- Executive Summary of Charcot-Marie-Tooth Disease
- Competitive Intelligence Analysis for Charcot-Marie-Tooth Disease
- Charcot-Marie-Tooth Disease: Market Overview at a Glance
- Charcot-Marie-Tooth Disease: Disease Background and Overview
- Patient Journey
- Charcot-Marie-Tooth Disease Epidemiology and Patient Population
- Treatment Algorithm, Current Treatment, and Medical Practices
- Charcot-Marie-Tooth Disease Unmet Needs
- Key Endpoints of Charcot-Marie-Tooth Disease Treatment
- Charcot-Marie-Tooth Disease Marketed Products
- Charcot-Marie-Tooth Disease Emerging Therapies
- Charcot-Marie-Tooth Disease: Seven Major Market Analysis
- Attribute analysis
- 7MM: Charcot-Marie-Tooth Disease Market Outlook
- Access and Reimbursement Overview of Charcot-Marie-Tooth Disease
- KOL Views
- Charcot-Marie-Tooth Disease Market Drivers
- Charcot-Marie-Tooth Disease Market Barriers
- DelveInsight Capabilities
- About DelveInsight
Know which therapy is expected to score the touchdown first @ Charcot Marrie Tooth Disease Market Drivers and Barriers
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