NS-089/NCNP-02 previously received Rare Pediatric Disease Designation from the FDA in June 2023 and Breakthrough Therapy Designation in July 2023
NS-089/NCNP-02 is an antisense nucleotide. (Credit: Dr. Edwin P. Ewing, Jr. from Wikimedia Commons)
NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
The FDA issues Orphan Drug Designations to support the development and evaluation of new treatments to prevent, diagnose, or treat a rare disease or condition.
NS-089/NCNP-02 previously received Rare Pediatric Disease Designation from the FDA in June 2023 and Breakthrough Therapy Designation in July 2023.
NS-089/NCNP-02 is an antisense nucleotide discovered through joint research between NS Pharma’s parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (Kodaira City; President, Kazuyuki Nakagome).
Clinical development of NS-089/NCNP-02 includes a planned Phase 2 study in the United States conducted by NS Pharma and a Phase 2 study conducted in Japan by Nippon Shinyaku. Additional details will be provided once the trials are ready to begin enrolling participants.
Source: Company Press Release