Press release
Charcot-Marie-Tooth (CMT) Disease Market to Reach US$ 1,166.98 Million by 2033 at 5.8% CAGR as Pipeline Advances and CMT1 Leadership Drive Progress | DataM Intelligence
AUSTIN, Texas and TOKYO -- According to DataM Intelligence, The Charcot-Marie-Tooth (CMT) Disease Market Size reached US$ 706.04 Million in 2024 (from US$ 670.21 Million in 2023) and is projected to reach US$ 1,166.98 Million by 2033, expanding at a CAGR of 5.8% during the forecast period 2025-2033. The Charcot-Marie-Tooth (CMT) Disease Market is transforming rare neurological care by combining rehabilitative therapies with emerging precision medicines for this group of demyelinating and axonal neuropathies.The shift from symptomatic relief alone to integrated diagnostic and therapeutic strategies reflects a broader transformation toward early genetic confirmation, functional improvement, slowed progression, and enhanced quality of life. Unlike conventional supportive approaches, modern solutions incorporate advanced orthotics, therapy protocols, and investigational candidates from companies like NMD Pharma A/S, ActioBio, InFlectis BioScience, HELIXMITH Co., Ltd., ENCell Corp., Addex Therapeutics, and Augustine Therapeutics targeting specific CMT mechanisms. This innovation is not just supportive; it is a paradigm shift in rare neuromuscular disorders, genetic testing, and specialized clinics.
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53 - Tables
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180 - Pages
Market Segmentation - Growth Anchors
By Disease Type
In 2024, CMT1 (primarily demyelinating, e.g., CMT1A due to PMP22 duplication) led with 52.42% revenue share, driven by highest prevalence and focus on myelin-targeted therapies. CMTX, CMT4, CMT2 (axonal), and Others follow, with growing interest in subtype-specific approaches.
By Treatment Type
Physical Therapy and Occupational Therapy dominate symptomatic management to maintain mobility, strength, and daily function. Orthopedic Devices (braces, orthotics, surgery aids) hold significant share for deformity correction and gait support. Others include pain management and emerging investigational drugs.
By End-User
Hospitals and Specialty Clinics account for major share due to multidisciplinary teams and access to diagnostics/therapy. Home Care Settings and Others (rehab centers) grow with demand for ongoing support.
Regional Insights: North America & Asia Pacific
North America
North America dominates the Charcot-Marie-Tooth (CMT) Disease Market with 44.37% revenue share in 2024, led by:
-Advanced genetic testing, high diagnosis rates, and strong orphan drug incentives.
-Robust clinical trial infrastructure and patient advocacy.
-Presence of key pipeline players like NMD Pharma A/S, ActioBio, and Augustine Therapeutics.
-Recent developments include FDA orphan designations and trial advancements in 2025.
Asia Pacific
Asia Pacific is the fastest-growing region, expected to expand at a CAGR of 6.1% over the forecast period, fueled by:
-Rising awareness, improving rare disease diagnostics in China, India, and Japan.
-Increasing healthcare investments and trial participation.
-Growing collaborations for gene/cell therapies.
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Industry Trends & 2025 Developments
-Pipeline Momentum: In 2025, NMD Pharma A/S received FDA Orphan Drug Designation for NMD670 (ClC-1 inhibitor) in January and completed enrollment in the Phase 2 SYNAPSE-CMT trial (results expected early 2026). Augustine Therapeutics dosed the first patient in Phase I for AGT-100216 (HDAC6 inhibitor) in May.
-Orphan Designations & Trials: ENCell Corp. secured FDA ODD for EN001 (stem cell therapy) in March; ActioBio dosed first participant in Phase 1 for ABS-0871 (TRPV4 inhibitor) for CMT2C in 2025.
-Therapy Focus: InFlectis BioScience advanced IFB-088 (icerguastat) in CMT1A; HELIXMITH progressed Engensis (VM202) gene therapy efforts.
-Awareness & Multidisciplinary Care: Enhanced genetic testing and rehab protocols gained traction in 2025 for better patient outcomes.
Competitive Landscape: Leaders Driving the Future
NMD Pharma A/S
NMD Pharma leads with NMD670, a novel ClC-1 inhibitor in Phase 2 for improving muscle function in CMT1/2.
ActioBio
ActioBio advances ABS-0871, a TRPV4 inhibitor targeting CMT2C, with Phase 1 underway.
InFlectis BioScience
InFlectis progresses IFB-088 (icerguastat) for CMT1A, focusing on neuroprotection.
HELIXMITH Co., Ltd.
HELIXMITH develops gene therapy approaches like Engensis (VM202) for CMT subtypes.
ENCell Corp.
ENCell pioneers EN001 stem cell therapy, with ODD and ongoing trials in CMT1A/1E.
Other Key Players
Addex Therapeutics and Augustine Therapeutics contribute through small-molecule HDAC6 inhibitors and other novel mechanisms.
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Strategic Outlook
The Charcot-Marie-Tooth (CMT) Disease Market is entering a transformative phase during 2025-2033. Four key forces will define its trajectory:
-Pipeline Innovation - Ion channel inhibitors, HDAC6 blockers, gene/cell therapies will drive potential disease-modifying options.
-Diagnostic Advances - Genetic subtype testing will enable earlier, targeted interventions.
-Rehabilitation Core - Physical/occupational therapy and orthotics will remain essential for symptom control.
-Regional Expansion - North America's dominance combined with Asia Pacific's fastest growth will accelerate global progress.
Conclusion
The Charcot-Marie-Tooth (CMT) Disease Market, projected to rise from US$ 706.04 Million in 2024 to US$ 1,166.98 Million by 2033, is central to rare neuropathy advancement, functional preservation, and emerging therapies. With North America holding 44.37% share and Asia Pacific setting the fastest CAGR of 6.1%, the sector offers compelling opportunities for neurologists, biotech firms, patients, and investors focused on inherited neuromuscular disorders.
Contact Us:
Sai Kiran
DataM Intelligence 4market Research LLP
USA: +1 877-441-4866
Email: Sai.k@datamintelligence.com
About DataM Intelligence
DataM Intelligence is a renowned provider of market research, delivering deep insights through pricing analysis, market share breakdowns, and competitive intelligence. The company specializes in strategic reports that guide businesses in high-growth sectors such as nutraceuticals and AI-driven health innovations.
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