Breakthrough Treatment for Duchenne Muscular Dystrophy Gets FDA Orphan Drug Designation

NS Pharma, Inc. today announced an incredible breakthrough for patients with Duchenne muscular dystrophy: The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NS-089/NCNP-02, an investigational candidate designed for exon 44 skipping therapy. This is a major step forward for improving patient outcomes and providing much needed hope to those living with this devastating condition.

NS-089/NCNP-02, an innovative treatment to prevent, diagnose, or treat a rare disease or condition, achieved two major milestones in June and July 2023: the Rare Pediatric Disease Designation from the FDA and the Breakthrough Therapy Designation. The designations were issued to support the development and evaluation of this potential new treatment, providing hope for patients and families dealing with rare diseases and conditions.

In a groundbreaking collaboration between NS Pharma’s parent company Nippon Shinyaku and the National Center of Neurology and Psychiatry (Kodaira City; President, Kazuyuki Nakagome), a significant discovery was made – a novel anti-sense nucleotide, NS-089/NCNP-02. This opens up new possibilities in the study of neurological conditions, and marks a major step towards better treatment for patients.

NS Pharma and Nippon Shinyaku are advancing the clinical development of NS-089/NCNP-02 with planned Phase 2 studies: one in the United States and one in Japan. Stay tuned for more information as these trials are readied for patient recruitment!

About Duchenne Muscular Dystrophy (Duchenne)

Duchenne is a devastating form of muscular dystrophy that primarily affects males. Early indications of Duchenne may include delayed ability to perform simple tasks such as sitting, standing or walking.

With the passage of time, patients with Duchenne experience progressive weakness and loss of their skeletal, cardiac, and respiratory muscles. Unfortunately, by adolescence, patients require the use of a wheelchair to move around and struggle with life-threatening cardiac and respiratory issues.

About NS Pharma, Inc.

NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., develops revolutionary dru gs and therapies with its globally recognized trademark, NS Pharma. With cutting-edge research and innovative ideas, NS Pharma continues to advance the health and wellbeing of people around the world.

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