by Charcot-Marie-Tooth disease, also known as hereditary motor and sensory neuropathy, is a inherited peripheral neuropathy that is characterized by progressive wasting and weakness of the muscles in the legs and hands. It is one of the most common inherited neurological disorders. Symptoms typically appear in the first two decades of life and can include high arched feet, leg deformities including hammertoes, decreased/absent tendon reflexes and weakness of the ankles/leg muscles. While there is currently no cure for Charcot-Marie-Tooth disease, medications and physiotherapy can help manage symptoms. Gene therapy and stem cell therapies are also emerging as promising treatment options.
The global Charcot-Marie-Tooth disease market is estimated to be valued at US$ 155 million in 2023 and is expected to exhibit a CAGR of 3.6% over the forecast period 2023-2033, as highlighted in a new report published by Coherent Market Insights.
Market key trends:
One of the major trends in the Charcot-Marie-Tooth disease market is increasing focus on gene therapy. Several pharmaceutical companies are conducting clinical trials on gene therapies for Charcot-Marie-Tooth disease. For instance, Audentes Therapeutics is conducting phase 1/2 clinical trial of AT342, an AAV8 gene therapy targeting defects in the GARS gene which causes a type of CMT. Initial safety and tolerability data from this trial was encouraging with no clinically significant safety issues reported. Similarly, Regenxbio is developing RGX 051, an AAV9 gene therapy targeting MPZ-associated CMT1B which is currently in preclinical stage of development. Another trend is growing research on stem cell therapy as a potential treatment approach. Mount Sinai researchers are exploring using mesenchymal stem cells derived from bone marrow which secrete neurotrophic factors to promote nerve regeneration in CMT.
Porter’s Analysis:
Threat of new entrants: Low, as developing treatment requires extensive research and regulatory approvals.
Bargaining power of buyers: Moderate, as there are few treatment options but patient population creates buyer power.
Bargaining power of suppliers: High, as key players have patents and expertise in rare disease therapeutics.
Threat of new substitutes: Low, as there are limited treatment options for Charcot-Marie-Tooth disease currently.
Competitive rivalry: High, as leading players are investing heavily in R&D to develop new drugs and gain market share.
Key Takeaways:
The Global Charcot-Marie-Tooth Disease Market Size is expected to witness high growth.
Regional analysis:
North America is currently dominating the market due to advanced healthcare facilities and growing R&D activities. However, Asia Pacific is expected to be the fastest growing region owing to improving healthcare infrastructure in emerging economies.
Key players related content comprises Key players related content: Key players operating in the Charcot-Marie-Tooth disease market are Pfizer, Adesto Therapeutics, RegeneRx Biopharmaceuticals, IceTherapeutics.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
