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Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S

WASHINGTON, June 1, 2023 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VCA-894A for the ...
BioWorld

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

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), ...
Healio

FDA grants orphan drug designation to RNA therapy for Charcot-Marie-Tooth disease type 1A

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Business Wire

ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease

EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial confirmed ...
KXAN

ReviR Therapeutics Receives Orphan Drug Designation from FDA for RTX-117 to Treat Charcot-Marie-Tooth Disease

BRISBANE, Calif. , Oct. 13, 2025 /PRNewswire/ -- ReviR Therapeutics, a biotech company focused on developing novel treatments for neurogenetic diseases, announced today that the U.S. Food and Drug ...