DelveInsight's "Charcot Marie Tooth Disease Pipeline Insight, 2025" report delivers extensive analysis on over 10 companies and more than 15 pipeline drugs in the Charcot Marie Tooth Disease treatment arena. It includes thorough profiles of Charcot Marie Tooth pipeline drugs, encompassing clinical and nonclinical development stages. Additionally, it evaluates Charcot Marie Tooth therapeutics by product category, development phase, administration method, and molecular structure. It also points out discontinued pipeline products in this domain.

Curious about the latest updates in the Charcot Marie Tooth Pipeline? Click here to explore the therapies and trials making headlines @ Charcot Marie Tooth Pipeline Outlook Report

Key Takeaways from the Charcot Marie Tooth Pipeline Report

• On October 23, 2025, NMD Pharma A/S disclosed a Phase 2a trial aimed at assessing the effectiveness, safety, and tolerability of NMD670 versus placebo given twice daily (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease types 1 and 2.

• DelveInsight's Charcot Marie Tooth Pipeline report illustrates a vibrant arena with over 10 active contributors developing more than 15 pipeline treatments for Charcot Marie Tooth care.

• The prominent Charcot Marie Tooth Companies include Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.

• Promising Charcot Marie Tooth Therapies include PXT3003, NMD670, MD1003, Engensis (VM202), Remibrutinib, Teriflunomide, Cladribine (MAVENCLAD Registered), and others.

Want to know which companies are leading innovation in Charcot Marie Tooth? Dive into the full pipeline insights @ Charcot Marie Tooth Clinical Trials Assessment 

Charcot Marie Tooth Overview

Charcot-Marie-Tooth disease (CMT) encompasses a set of genetic neurological conditions impacting peripheral nerves, responsible for limb movement and sensation. Identified by three doctors in 1886, CMT is also termed hereditary motor and sensory neuropathy (HMSN). It's among the most prevalent inherited neurological issues, impacting roughly 1 in 2,500 individuals. CMT features gradual muscle weakening and shrinkage, mainly in the legs and feet, and later in the hands and arms.

Charcot Marie Tooth Disease Emerging Drugs

• PXT3003: Pharnext SA

PXT3003 is an innovative fixed-dose synergistic blend of baclofen, naltrexone, and sorbitol, prepared as an oral solution taken twice daily. The three components of PXT3003 are chosen to reduce the excess production of PMP22 protein, enhancing neuronal signaling in impaired peripheral nerves central to the disease's pathology. PXT3003 may also benefit other motor unit cell types like axons (direct safeguarding), neuromuscular junctions, or muscle cells. It is currently in Phase III development for Charcot Marie Tooth disease type 1A.

• IFB-088: InFlectis BioScience

IFB-088 is an orally administered small molecule that penetrates the blood-brain barrier, a protective layer shielding the brain from harmful substances in the bloodstream. IFB-088 acts as an inhibitor of specific glutamate receptors. These receptors contribute to excitotoxicity, where neurons become excessively stimulated and damaged. IFB-088 holds promise for various demyelinating subtypes, including CMT 1A, CMT 1B, and CMT 1E, affecting 150,000 people in the U.S. and Europe. The Company is preparing a Phase II clinical trial in the United States and Europe. The drug is in Phase II trials for Charcot Marie Tooth disease.

If you're tracking ongoing Charcot Marie Tooth Clinical trials, this press release is a must-read. Tap to see the breakthroughs @ Charcot Marie Tooth Treatment Drugs 

Charcot Marie Tooth Companies

Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.

The Charcot Marie Tooth Pipeline report provides insights into:-

• The report furnishes comprehensive details on organizations advancing therapies for Charcot Marie Tooth, including the total treatments each company is developing.

• It examines various therapeutic candidates categorized into initial, intermediate, and advanced development stages for Charcot Marie Tooth management.

• Charcot Marie Tooth Companies are engaged in precision therapeutics with ongoing and paused (inactive or halted) initiatives.

• Charcot Marie Tooth Drugs in progress, sorted by development phase, delivery route, target receptor, standalone or combined therapy, unique action mechanisms, and molecular category.

• Comprehensive review of partnerships (corporate alliances and academic ties), licensing deals, and funding information to drive future growth in the Charcot Marie Tooth market.

Charcot Marie Tooth Disease pipeline report assesses the therapeutic potential of pipeline drugs by Route of Administration. Products are grouped under different ROAs, including

• Intravenous

• Subcutaneous

• Oral

• Intramuscular

Charcot Marie Tooth Products have been categorized under various Molecule types such as

• Monoclonal antibody

• Small molecule

• Peptide

From emerging drug candidates to competitive intelligence, the Charcot Marie Tooth Pipeline Report covers it all - check it out now @ Charcot Marie Tooth Market Drivers and Barriers, and Future Perspectives 

Scope of the Charcot Marie Tooth Pipeline Report

• Coverage- Global

• Charcot Marie Tooth Companies- Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.

• Charcot Marie Tooth Therapies- PXT3003, NMD670, MD1003, Engensis (VM202), Remibrutinib, Teriflunomide, Cladribine (MAVENCLAD Registered), and others.

• Charcot Marie Tooth Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination

• Charcot Marie Tooth Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Stay ahead in Healthcare Research - discover what's next for the Charcot Marie Tooth Treatment landscape in this detailed analysis @ Charcot Marie Tooth Emerging Drugs and Major Players 

Table of Content

• Introduction

• Executive Summary

• Charcot Marie Tooth Disease: Overview

• Pipeline Therapeutics

• Therapeutic Assessment

• Charcot Marie Tooth Disease- DelveInsight's Analytical Perspective

• Late Stage Products (Phase III)

• PXT3003: Pharnext SA

• Drug profiles in the detailed report.....

• Mid Stage Products (Phase II)

• Drug Name: Company Name

• Drug profiles in the detailed report.....

• Early Stage Products (Phase I)

• IFB-088: InFlectis BioScience

• Drug profiles in the detailed report.....

• Preclinical and Discovery Stage Products

• Drug Name: Company Name

• Drug profiles in the detailed report.....

• Inactive Products

• Charcot Marie Tooth Disease Key Companies

• Charcot Marie Tooth Disease Key Products

• Charcot Marie Tooth Disease- Unmet Needs

• Charcot Marie Tooth Disease- Market Drivers and Barriers

• Charcot Marie Tooth Disease- Future Perspectives and Conclusion

• Charcot Marie Tooth Disease Analyst Views

• Charcot Marie Tooth Disease Key Companies

• Appendix

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