Global CMT Disease Market Valued at USD 706.04 Mn in 2024 | Growth Outlook to 2033

AUSTIN, TX, UNITED STATES, October 28, 2025 /EINPresswire.com/ -- Charcot-Marie-Tooth (CMT) disease market Overview

Charcot-Marie-Tooth (CMT) disease, one of the most prevalent inherited neurological disorders, affects approximately 1 in 2,500 individuals globally. Characterized by progressive muscle weakness and sensory loss, CMT encompasses a diverse spectrum of subtypes, each with unique genetic underpinnings and clinical manifestations. The disease primarily impacts the peripheral nervous system, leading to symptoms such as foot deformities, gait abnormalities, and muscle atrophy.

Market Size and Forecast

The global Charcot-Marie-Tooth (CMT) disease industry was valued at USD 670.21 million in 2023 and increased to USD 706.04 million in 2024. The market is projected to reach USD 1,166.98 million by 2033, expanding at a compound annual growth rate (CAGR) of 5.8% between 2025 and 2033. This growth is driven by several factors, including advancements in diagnostic technologies, increased awareness, and a robust pipeline of therapeutic candidates.

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Key Market Highlights

North America held the dominant position in the Charcot-Marie-Tooth (CMT) disease market, accounting for the largest revenue share of 44.37% in 2024, supported by advanced healthcare infrastructure and strong research initiatives.

The Asia Pacific region emerged as the fastest-growing market, projected to register the highest CAGR of 6.1% during the forecast period, driven by improving diagnostic access and increasing disease awareness.

By disease type, the CMT1 segment remained the market leader, capturing a 52.42% revenue share in 2024, owing to its higher prevalence and better-established diagnostic capabilities compared to other subtypes.

Key Market Drivers

Advancements in Genetic Testing: The advent of next-generation sequencing and other genetic diagnostic tools has revolutionized the identification of CMT subtypes. Early and accurate diagnosis enables personalized treatment approaches, improving patient outcomes.

Growing Therapeutic Pipeline: Historically, treatment options for CMT have been limited to symptomatic management. However, recent years have seen a surge in the development of disease-modifying therapies. Notable candidates include IFB-088 by InFlectis BioScience and EN001 by ENCell, both targeting specific genetic mutations associated with CMT. Additionally, NMD670, developed by NMD Pharma, is undergoing Phase 2 clinical trials for its efficacy in treating CMT1 and CMT2 subtypes.

Increased Awareness and Advocacy: Organizations such as the Charcot-Marie-Tooth Association (CMTA) have played a pivotal role in raising awareness about CMT, leading to improved diagnosis rates and patient support. The CMTA's STAR program, encompassing over 50 active research initiatives, underscores the collaborative efforts to accelerate therapeutic development.

Key Players:

1. NMD PHARMA A/S
2. ActioBio
3. InFlectis BioScience
4. HELIXMITH Co., Ltd.
5. ENCell Corp.
6. Addex Therapeutics
7. Augustine Therapeutics

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Regional Insights: Charcot-Marie-Tooth (CMT) Disease Market

North America:

North America led the global CMT disease market in 2024, holding a 44.37% share, and is expected to maintain dominance through the forecast period. The region’s leadership is driven by a strong research ecosystem, high disease prevalence, and early adoption of innovative therapies. The U.S., with an estimated 125,000–150,000 diagnosed cases, benefits from advanced genetic testing facilities, specialized neuromuscular centers, and FDA incentives like Orphan Drug and Fast Track programs. Notably, in January 2025, NMD Pharma received FDA Orphan Drug Designation for its therapy NMD670, while Actio Biosciences began Phase 1 trials of ABS-0871 for CMT2C reflecting the region’s active clinical pipeline.

Asia-Pacific:

The Asia-Pacific (APAC) region is emerging as the fastest-growing market, projected to expand at a CAGR of 6.1%. Growth is driven by rising healthcare investments, expanding genetic testing capabilities, and greater clinician awareness. Countries like China, Japan, and India are building rare disease frameworks, advancing gene therapy infrastructure, and establishing favorable regulatory pathways for orphan drugs. While no CMT-specific therapies have yet launched in APAC, the region plays an increasingly vital role in global clinical trials and early-access initiatives, positioning it for rapid adoption once new treatments receive approval.

Europe:

Europe is experiencing steady growth supported by strong regulatory frameworks, early diagnosis, and increasing research collaborations. The European Medicines Agency (EMA) continues to grant orphan designations and accelerate rare disease approvals, stimulating R&D investments. A shift toward multidisciplinary care combining physiotherapy, orthotic devices, and genetic counseling is improving patient quality of life. With growing clinical activity and biotech partnerships, Europe is set to remain a key growth hub for CMT therapeutics in the next decade.

Market Segmentation

Disease Type: The CMT1 segment dominated the Charcot-Marie-Tooth (CMT) disease market in 2024, accounting for 52.42% of the total market share, primarily due to its higher prevalence and well-defined diagnostic framework.

By Disease Type: (CMT1, CMTX, CMT4, CMT2, and Others)

By Treatment Type: (Physical Therapy, Occupational Therapy, Orthopedic Devices, and Others)

By End-User: (Hospitals, Specialty Clinics, Home Care Settings, and Others)

By Region: (North America, Europe, Asia-Pacific, South America and the Middle East & Africa)

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Strategic Recommendations

Investment in Research and Development: Companies should prioritize investment in R&D to develop novel therapies targeting specific genetic mutations associated with CMT. Collaborations with academic institutions and research organizations can enhance innovation and expedite the development process.

Expansion of Diagnostic Services: Establishing specialized centers for genetic testing and counseling can facilitate early diagnosis and personalized treatment plans, improving patient outcomes.

Patient-Centric Approaches: Focusing on patient education, support services, and accessibility to treatments can enhance the quality of life for individuals affected by CMT.

Conclusion

The CMT disease market is poised for significant growth, driven by advancements in diagnostic technologies, a burgeoning therapeutic pipeline, and increased awareness and advocacy efforts. By focusing on research and development, expanding diagnostic services, and adopting patient-centric approaches, stakeholders can contribute to improving the lives of individuals affected by this debilitating condition.

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