Vanda Pharmaceuticals announced that the FDA has approved the Investigational New Drug, or IND, application to evaluate VCA-894A for the treatment of a patient with Charcot-Marie-Tooth disease, axonal, type 2S – CMT2S -, caused by cryptic splice site variants within the IGHMBP2 gene. CMT2S is a rare subtype of Charcot-Marie-Tooth disease, an inherited peripheral neuropathy for which there is no available treatment. CMT2S is characterized by slowly progressive distal muscle weakness and atrophy, affecting the upper and lower limbs in a child’s first decade of life. The estimated overall prevalence of CMT is 1 in 2,500 individuals. The prevalence of the CMT2S variant is estimated to be less than 1 in 1,000,000 worldwide.
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