FDA grants rare paediatric designation to Vanda’s CMT2S therapy

FDA grants RPDD to Vanda’s investigational CMT2S therapy - BioTuesdays

Vanda Pharmaceuticals (NASDAQ: VNDA) has announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational antisense oligonucleotide therapy—VCA-894A—for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). CMT2S is a rare, serious, and progressive inherited neurological disorder primarily affecting individuals from birth through 18 years of age. In a statement, Mihael H. Polymeropoulos, MD, pr…

FDA grants rare paediatric designation to Vanda’s CMT2S therapy

The FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics granted this status to the therapy. CMT2S is a rare, inherited neurological condition that progressively causes The post FDA grants rare paediatric designation to Vanda’s CMT2S therapy appeared first on Pharmaceutical Business review.