Vanda's ASO VCA-894A awarded US orphan drug designation for Charcot-Marie-Tooth disease type 2S

June 2, 2023

Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2.

BioWorld Science Neurology/psychiatric FDA Orphan drug

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