Among the subtype of CMT, Type 1 is considered the most common category representing approximately ~75% of all CMT cases, and the prevalence of CMT Type 2 on the other hand ranges from 10-15 % of all CMT cases and comprises the second major subtype of CMT.
Comprehensive insight on patient segmentation based on types and subtypes (CMT1, CMT1A, CMT1B, CMT2, CMTx, and others types), Coexisting conditions has been provided into the epidemiology (Incidence and Prevalence) section of the Charcot-Marie-Tooth and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China.
The pipeline for Charcot–Marie–Tooth (CMT) is intense and di-verse MoAs are under evaluation. Key MoAs include GABA B receptor agonist, Opioid mu receptor antagonist, Opioid kappa receptor antagonist (PXT3003; Pharnext), TGF-beta inhibitors(ACE-083; Acceleron), and ATP production of against axonal degeneration & activating acetyl-CoA carboxylase (MD1003; MedDay), IFB-088 from InFlectis Biosciences is another promising drug candidate currently under development in Phase-I stage of development. In June 2014, European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to PXT-3003 for the treatment of Charcot-Marie-Tooth dis-ease type 1A (CMT 1A) and again in 2018 followed by Fast track designation in Feb 2019. PXT3003 was granted Priority Review for Charcot-Marie-Tooth Type 1A disease (CMT1A) by the China Food and Drug Administration (CFDA).
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