FDA grants rare paediatric designation to Vanda’s CMT2S therapy

The US FDA has granted rare paediatric disease designation to Vanda Pharmaceuticals’ VCA-894A, an investigational antisense oligonucleotide therapy for CMT2S. The status was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics. CMT2S is a rare, inherited neurological condition that progressively causes motor function loss and muscle weakness. Credit: SkazovD / Shutterstock.com. 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). The FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics granted this…

Source: Pharmaceutical Technology