FDA grants Vanda rare pediatric disease tag for CMT2S therapy

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Go back to FDA grants Vanda rare pediatric disease tag for CMT2S therapy

Vanda Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Investigational Therapy for Charcot-Marie-Tooth Disease Type 2S

July 7, 2026 4:30 PM EDT

WASHINGTON, July 7, 2026 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to VCA-894A, Vanda's investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disorder. The... More