Charcot Marie Tooth Disease Market on Track for Major Expansion by 2034, According to DelveInsight | DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharma

The Key Charcot Marie Tooth Disease Companies in the market include - Pharnext, DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharmaceutical, ENCell, HELIXMITH, Pharnext SA, MedDay Pharmaceuticals, and others.

DelveInsight's "Charcot Marie Tooth Disease Market Insights, Epidemiology, and Market Forecast-2034 report offers 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, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Charcot Marie Tooth Disease Market Report:

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The Charcot Marie Tooth Disease market size was valued approximately USD 58 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034).

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In October 2025, ReviR Therapeutics, a biotechnology company dedicated to developing innovative therapies for neurogenetic disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead candidate, RTX-117, for the treatment of Charcot-Marie-Tooth disease (CMT). The company plans to initiate a Phase 1 trial in healthy volunteers in early 2026 to assess safety, tolerability, and pharmacokinetics, followed by a Phase Ib/IIa study in CMT patients.

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In June 2025, Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) announced that the first patient has been dosed in its first-in-human clinical trial evaluating VCA-894A, an antisense oligonucleotide (ASO) therapy, for a rare IGHMBP2 gene variant causing Charcot-Marie-Tooth disease Type 2S (CMT2S). The patient, diagnosed at age five, has this rare inherited neuromuscular disorder, which progressively causes muscle weakness and loss of motor function. CMT2S affects fewer than 1 in 1,000,000 people globally, with its severity and symptoms varying based on the diverse genetic variants linked to CMT disease.

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In May 2025, Augustine Therapeutics NV ("Augustine"), a biotechnology company developing novel therapies for neuromuscular, neurodegenerative, and cardio-metabolic diseases by inhibiting the cytosolic Histone DeACetylase 6 (HDAC6) enzyme, announced that the first patient has been dosed in its Phase I clinical trial of lead candidate AGT-100216, the first peripherally restricted, selective HDAC6 inhibitor.

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In March 2025, Actio Biosciences, a clinical-stage biotechnology company applying its one-to-many approach in genetics and precision medicine to develop therapies targeting shared underlying biology in rare and common diseases, announced that the first participant has been dosed in a Phase 1 healthy volunteer trial of its lead candidate, ABS-0871. This TRPV4 inhibitor is being developed for the treatment of TRPV4-positive Charcot-Marie-Tooth disease subtype 2C (CMT2C), a severe peripheral nerve disorder.

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In May 2025, Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company focused on developing transformative therapies for rare diseases, presented full 12-month clinical results along with new topline data at 18 and 24 months from the INSPIRE Phase 2/3 trial of govorestat (AT-007) for treating Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The data were shared in an oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, held May 17-20, 2025, in Edinburgh, Scotland.

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In March 2025, ENCell, a prominent biopharmaceutical company specializing in cell and gene therapy CDMO services and innovative drug development, announced that its investigational drug EN001 has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for treating Charcot-Marie-Tooth disease (CMT). CMT is an inherited neuromuscular disorder that leads to progressive muscle weakness and deformities in the hands and feet, and in severe cases, may result in vision and hearing impairments.

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In January 2025, Biotechnology company NMD Pharma announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its Charcot-Marie-Tooth (CMT) disease therapy, NMD670. Previously, NMD670 had received ODD from the FDA for generalized myasthenia gravis in September 2022. NMD670 is a small molecule inhibitor targeting the skeletal muscle-specific chloride ion channel CIC-1. In November 2024, NMD Pharma initiated the Phase 2 SYNAPSE-CMT trial, evaluating a twice-daily oral dose of NMD670 over 21 days in 80 adult patients with genetically confirmed CMT1 or CMT2 subtypes. CMT is a hereditary neuropathy affecting an estimated 136,000 individuals in the US and three million globally, causing muscle weakness, fatigue, and sensory deficits.

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In 2023, Germany held the largest market share among the EU4 and the UK, with an estimated value of around USD 4 million, followed by France and the United Kingdom.

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In 2023, Japan's Charcot-Marie-Tooth Disease market was valued at approximately USD 2 million, with projections indicating growth throughout the forecast period (2020-2034).

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In 2023, the total prevalent population of Charcot-Marie-Tooth (CMT) disease in the 7MM was approximately 268 thousand. These cases are anticipated to rise over the study period from 2020 to 2034.

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In 2023, the total diagnosed prevalent population of Charcot-Marie-Tooth (CMT) disease in the 7MM was approximately 190 thousand, with the US having the largest number of cases at around 100 thousand.

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In 2023, among the EU4 and the UK, Germany had the highest diagnosed prevalent population of approximately 22 thousand cases, followed by France with around 19 thousand and the United Kingdom with about 18 thousand. Spain had the lowest diagnosed prevalent population at approximately 10 thousand cases.

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In the epidemiology model of Charcot-Marie-Tooth (CMT), various age groups are categorized as 0-18 years, 19-40 years, 41-60 years, and 45+ years. In the US, the highest number of cases, nearly 34 thousand, were observed in the 41-60 year age group in 2023.

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In 2023, Charcot-Marie-Tooth (CMT) affected a higher number of males. Of the total diagnosed prevalent population of CMT in Japan, nearly 6 thousand cases were male, compared to approximately 3 thousand cases in females.

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Key Charcot Marie Tooth Disease Companies: Pharnext, DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharmaceutical, ENCell, HELIXMITH, Pharnext SA, MedDay Pharmaceuticals, and others

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Key Charcot Marie Tooth Disease Therapies: PXT3003, DTx-1252, HDAC6i, Research programme: GABAB PAM, IFB-088, CKD-510, EN001, Engensis, PXT3003, MD1003, and others

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The Charcot Marie Tooth Disease market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Charcot Marie Tooth Disease pipeline products will significantly revolutionize the Charcot Marie Tooth Disease market dynamics.

Charcot Marie Tooth Disease Overview

Charcot-Marie-Tooth (CMT) disease is a group of inherited neurological disorders that affect the peripheral nerves, which connect the brain and spinal cord to the muscles and sensory organs. This progressive condition damages the myelin sheath (protective covering of nerves) or the nerve fibers themselves, leading to weakness, muscle wasting, and reduced sensation in the legs, feet, and sometimes the hands. Symptoms typically appear in adolescence or early adulthood and may include difficulty walking, foot deformities, and a loss of fine motor skills. While there is no cure for CMT, physical therapy, occupational therapy, and orthopedic devices can help manage symptoms and improve quality of life.

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Charcot Marie Tooth Disease Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Charcot Marie Tooth Disease Epidemiology Segmentation:

The Charcot Marie Tooth Disease market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalent Cases of Charcot Marie Tooth Disease in the 7MM

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Diagnosed Prevalent Cases of Charcot Marie Tooth Disease in the 7MM

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Gender-specific Diagnosed Prevalent Cases of Charco Marie Tooth Disease in the 7MM

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Age-specific Diagnosed Prevalent Cases of Charcot Marie Tooth Disease in the 7MM

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Type-specific Diagnosed Prevalent Cases of Charco Marie Tooth Disease in the 7MM

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Charcot Marie Tooth Disease Drugs Uptake and Pipeline Development Activities

The drugs uptake 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 during the study period. The analysis covers Charcot Marie Tooth Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Charcot Marie Tooth Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Charcot Marie Tooth Disease Therapies and Key Companies

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PXT3003: Pharnext

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DTx-1252: DTx Pharma

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HDAC6i: Augustine therapeutics

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Research programme: GABAB PAM: Addex Therapeutics

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IFB-088: InFlectis BioScience

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CKD-510: Chong Kun Dang pharmaceutical

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EN001: ENCell

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Engensis: HELIXMITH

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PXT3003: Pharnext SA

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MD1003: MedDay Pharmaceuticals

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Charcot Marie Tooth Disease Market Strengths

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The increasingly prevalent population worldwide is likely to cause a surge in treatment options, and consequently, the market will witness a boost in upcoming years.

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The shortfall of approved therapies might provide goodspace for emerging therapies in the market.

Charcot Marie Tooth Disease Market Opportunities

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The unavailability of approved treatment options offers a great opportunity for the investment and development of novel therapies.

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Medications with better safety and effectiveness, which provide an optimum cure, are the current unmet need of theCMT disease market

Scope of the Charcot Marie Tooth Disease Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Charcot Marie Tooth Disease Companies: Pharnext, DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharmaceutical, ENCell, HELIXMITH, Pharnext SA, MedDay Pharmaceuticals, and others

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Key Charcot Marie Tooth Disease Therapies: PXT3003, DTx-1252, HDAC6i, Research programme: GABAB PAM, IFB-088, CKD-510, EN001, Engensis, PXT3003, MD1003, and others

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Charcot Marie Tooth Disease Therapeutic Assessment: Charcot Marie Tooth Disease current marketed and Charcot Marie Tooth Disease emerging therapies

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Charcot Marie Tooth Disease Market Dynamics: Charcot Marie Tooth Disease market drivers and Charcot Marie Tooth Disease market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Charcot Marie Tooth Disease Unmet Needs, KOL's views, Analyst's views, Charcot Marie Tooth Disease Market Access and Reimbursement

To know more about Charcot Marie Tooth Disease companies working in the treatment market, visit @ Charcot Marie Tooth Disease Clinical Trials and Therapeutic Assessment [https://www.delveinsight.com/sample-request/charcot-marie-tooth-disease-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Charcot Marie Tooth Disease Market Report Introduction

2. Executive Summary for Charcot Marie Tooth Disease

3. SWOT analysis of Charcot Marie Tooth Disease

4. Charcot Marie Tooth Disease Patient Share (%) Overview at a Glance

5. Charcot Marie Tooth Disease Market Overview at a Glance

6. Charcot Marie Tooth Disease Disease Background and Overview

7. Charcot Marie Tooth Disease Epidemiology and Patient Population

8. Country-Specific Patient Population of Charcot Marie Tooth Disease

9. Charcot Marie Tooth Disease Current Treatment and Medical Practices

10. Charcot Marie Tooth Disease Unmet Needs

11. Charcot Marie Tooth Disease Emerging Therapies

12. Charcot Marie Tooth Disease Market Outlook

13. Country-Wise Charcot Marie Tooth Disease Market Analysis (2020-2034)

14. Charcot Marie Tooth Disease Market Access and Reimbursement of Therapies

15. Charcot Marie Tooth Disease Market Drivers

16. Charcot Marie Tooth Disease Market Barriers

17. Charcot Marie Tooth Disease Appendix

18. Charcot Marie Tooth Disease Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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