Posted by Michael Wonder on 07 Jul 2023
FDA grants rare paediatric disease designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma's parent company, Nippon Shinyaku, and the National Center for Psychiatry and Neurological Medicine (Kodaira City, President: Kazuyuki Nakagome).
