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July 8, 2026

FDA grants rare paediatric designation to Vanda’s CMT2S therapy

The US Food and Drug Administration (FDA) has granted rare paediatric disease designation to Vanda Pharmaceuticals’ VCA-894A, an investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).

FDA grants rare paediatric designation to Vanda’s CMT2S therapy