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NS Pharma: FDA Grants Orphan Drug Designation to NS-089/NCNP-02

August 9, 2023

NS Pharma has announced that 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.

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.