NS Pharma, Inc. announced today the U.S. Food & Drug Administration  has granted Rare Pediatric Disease Designation to NS-089/NCNP-02 (brogidirsen) an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

The FDA's Rare Pediatric Disease Designation is granted for treatments intended for serious or life-threatening diseases that affect children under the age of 18 and less than 200,000 patients in the U.S.

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).

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:nspharma.com

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