FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment

Duchenne muscular dystrophy

The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS Pharma Inc.’s NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

The breakthrough designation is based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan. In June 2023, NS-089/NCNP-02 was granted Rare Pediatric Disease Designation by the FDA.

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. Additional details will be provided once the trials are ready to begin enrolling participants.

About Duchenne muscular dystrophy

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. Duchenne causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. 

Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.

NS Pharma pipeline

NS Pharma has several other Duchenne muscular dystrophy treatments in development. Its CAP-1002 cell therapy treatment and NS-065/NCNP-01 (viltolarsen) are in phase 3 trials, while its selective JAK2 inhibitor, NS-018, is in phase 2. There are four other potential treatments at the preclinical stage. 

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