The global Charcot Marie Tooth Disease Market is estimated to be valued at US$ 1208.92 Mn in 2023 and is expected to exhibit a CAGR of 23% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Overview:
Charcot Marie Tooth disease (CMT) is a genetic disorder that affects the peripheral nervous system, specifically the peripheral nerves located outside of the brain and spinal cord. The disease causes damage to the insulation covering nerve fibers known as myelin sheath or the nerve fibers themselves resulting in impaired nerve signaling between the limbs and brain. Clinical symptoms include muscle weakness and wasting in the legs and arms, reduced or absent tendon reflexes, and deformities of the foot and lower legs. At present, there is no cure for CMT and treatment involves pain relief, physical or occupational therapy, leg braces, and in severe cases, surgery. The global Charcot Marie Tooth disease market consists of drug candidates in different stages of clinical trials.
Market key trends:
One of the key trends in the Charcot Marie Tooth disease market is rising research into developing gene therapies. Several pharmaceutical companies are exploring ways to deliver genes that can potentially replace faulty genes causing CMT. For instance, Phase 1/2 clinical trial is evaluating Pharnext’s PXT3003, a combination of baclofen, naltrexone and sorbitol, to treat CMT1A. Increased funding from government and private organizations for CMT disease research is also boosting the market growth. Moreover, growing CMT disease registries and databases are helping researchers understand disease progression and outcomes, thereby facilitating drug development process. However, high development costs associated with rare disease therapies continue to remain a major challenge.
Porter’s Analysis
Threat of new entrants: The threat of new entrants is moderate as the Pharmaceutical industry requires high R&D investment and regulatory approvals. However, increasing awareness and demand provide growth opportunities.
Bargaining power of buyers: The bargaining power of buyers is moderate due to the presence of alternative treatment options. Patients are also sensitive to pricing.
Bargaining power of suppliers: Suppliers have low bargaining power due to the availability of substitute raw materials. Industry concentration is also low.
Threat of new substitutes: The threat of new substitutes is high as research is ongoing for developing newer therapies and treatment options.
Competitive rivalry: The competitive rivalry is high owing to the presence of many pharmaceutical companies.
Key Takeaways
The global Charcot Marie Tooth Disease market is expected to witness high growth, exhibiting CAGR of 23.% over the forecast period, due to increasing research and development activity in orphan drugs by pharma companies.
Regional analysis: North America dominated the market in 2022 owing to high awareness levels and advanced healthcare infrastructure. However, Asia Pacific is expected to grow at the fastest pace during the forecast period due to rising healthcare expenditure and patient pool in China and India.
Key players: Key players operating in the Charcot Marie Tooth Disease market are Addex Therapeutics Ltd., Affectis Pharmaceuticals AG, Genzyme Corp, Lead Discovery Center GmbH, Pharnext SA. Acceleron Pharma is a leading company in orphan drugs and conducted phase 3 trials for CMT drug candidates. Neurogene Inc. is also conducting clinical trials to develop promising therapies.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it