X-linked Charcot-Marie-Tooth Disease Market

Market Overview:

The 7 major x-linked charcot-marie-tooth disease markets reached a value of USD 555.1 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 3,554.1 Million by 2033, exhibiting a growth rate (CAGR) of 18.39% during 2025-2035.

The x-linked charcot-marie-tooth disease market has been comprehensively analyzed in IMARC's new report titled "X-linked Charcot-Marie-Tooth Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". X-linked Charcot-Marie-Tooth disease (CMTX) is an inherited peripheral neuropathy resulting from mutations in the GJB1 gene, which codes for connexin 32, an essential protein for Schwann cell function and myelination. It mainly occurs in males because of the X-linked pattern of inheritance, whereas female carriers can have milder symptoms. It is distinguished by distal nerve degeneration that affects peripheral nerves progressively, resulting in weakness of the muscles, loss of sensation, deformities like high arch (pes cavus) and hammertoes of the feet, and weakened reflexes. Its onset usually develops during childhood or early adulthood with aggravation over time, ultimately resulting in difficulties with walking, constant falls, and fine motor disability. Its advanced forms will lead to wheelchair dependency. Diagnosis of CMTX includes clinical assessment, family history, electromyography (EMG), and nerve conduction studies (NCS) to identify nerve signal abnormalities. Genetic testing is important in the confirmation of GJB1 mutations. Further, newer imaging methods like magnetic resonance neurography are being investigated to evaluate the extent of nerve damage and disease progression effectively.

The increasing realization of inherited neuropathies and wider availability of genetic testing are key drivers for the X-linked Charcot-Marie-Tooth disease market. Precise and early genetic diagnosis is crucial for disease management and counseling of patients. The market is undergoing a growing interest in gene therapy and RNA-based treatments, which seek to correct mutations in GJB1 or modulate the course of the disease. Novel small-molecule treatments for targeting connexin dysfunction and neuroprotective therapies aimed at postponing nerve degeneration are in development. The promise of disease-modifying treatments, including antisense oligonucleotides and CRISPR gene editing, is very promising for attacking the root cause of the condition instead of symptomatic management. Furthermore, technological advances in neurorehabilitation, such as functional electrical stimulation (FES) and individually tailored physiotherapy programs, are enhancing mobility and patient outcomes. The increasing use of assistive devices, including ankle-foot orthoses and wearable exoskeletons, is also further improving the quality of life of CMTX patients. With the expanding pipeline for novel therapies, regulatory approvals and investment in clinical trials are likely to fuel market growth.

IMARC Group's new report provides an exhaustive analysis of the x-linked charcot-marie-tooth disease market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the x-linked charcot-marie-tooth disease market in any manner.

Recent Developments:

• In January 2025, the FDA granted orphan drug status to NMD670 for CMT, targeting ClC-1 channels to enhance muscle function. A Phase 2 trial is currently in progress.
• In September 2023, researchers secured funding to develop a nanoparticle-based gene therapy for CMT1X, offering a precise, non-viral approach to treating Schwann cell dysfunction.

Key Highlights:

• CMTX represents 10-15% of CMT cases, affecting approximately 1 in 100,000 individuals, making it the second most common subtype.
• Mutations in X-linked genes, particularly GJB1, cause CMTX, with six known subtypes impacting nerve function and myelin integrity.
• CMTX follows an X-linked dominant inheritance; males experience more severe symptoms, while females may have milder or asymptomatic presentations.
• Progressive muscle weakness, sensory loss, foot deformities, and reduced reflexes characterize CMTX, primarily affecting distal limb function.
• Diagnosis involves clinical exams, nerve studies, and genetic testing. Treatment focuses on rehabilitation, orthopedic support, and symptom management.

Drugs:

VCA-894A, which has been developed by OliPass and Vanda Pharmaceuticals, is an experimental treatment for X-linked Charcot-Marie-Tooth disease (CMTX). The new treatment is designed to target the root genetic pathways to reverse disease progression. VCA-894A, which is under development, is focused on enhancing nerve function and movement, providing promise of therapeutic effectiveness for CMTX patients.

NMD670 is NMD Pharma's experimental oral treatment for the skeletal muscle-specific chloride ion channel ClC-1. A drug intended to treat Charcot-Marie-Tooth disease (CMT), it increases responsiveness of the muscle, enhancing neuromuscular transmission and function restoration. Under Phase 2 trials, NMD670 has been granted FDA orphan drug status for CMT treatment.

IFB-088 is an orally accessible small molecule discovered by InFlectis BioScience for the treatment of Charcot-Marie-Tooth disease. IFB-088 targets the Integrated Stress Response, assisting in correct protein folding and nerve function. Preclinical research has indicated that IFB-088 enhances neuropathy in CMT1A and CMT1B mouse models. The European Medicines Agency gave orphan designation for the treatment of Charcot-Marie-Tooth disease to it.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the x-linked charcot-marie-tooth disease market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the x-linked charcot-marie-tooth disease market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current x-linked charcot-marie-tooth disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the x-linked charcot-marie-tooth disease market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the x-linked charcot-marie-tooth disease market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the x-linked charcot-marie-tooth disease market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of x-linked charcot-marie-tooth disease across the seven major markets?
• What is the number of prevalent cases (​2019-2035) of x-linked charcot-marie-tooth disease by age across the seven major markets?
• What is the number of prevalent cases (2019-2035​) of x-linked charcot-marie-tooth disease by gender across the seven major markets?
• How many patients are diagnosed (2019-2035) with x-linked charcot-marie-tooth disease across the seven major markets?
• What is the size of the x-linked charcot-marie-tooth disease patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of x-linked charcot-marie-tooth disease?
• What will be the growth rate of patients across the seven major markets?

X-linked Charcot-Marie-Tooth Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for x-linked charcot-marie-tooth disease drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the x-linked charcot-marie-tooth disease market?
• What are the key regulatory events related to the x-linked charcot-marie-tooth disease market?
• What is the structure of clinical trial landscape by status related to the x-linked charcot-marie-tooth disease market?
• What is the structure of clinical trial landscape by phase related to the x-linked charcot-marie-tooth disease market?
• What is the structure of clinical trial landscape by route of administration related to the x-linked charcot-marie-tooth disease market?