In a recently published press release, NS Pharma announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to NS-051/NCNP-04, an experimental therapy for Duchenne muscular dystrophy (DMD).
The Orphan Drug designation is intended to encourage the development of therapies for rare conditions that affect fewer than 200,000 people in the United States.
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NS-051/NCNP-04 is an antisense oligonucleotide codiscovered by the National Center of Neurology and Psychiatry and Nippon Shinyaku. The drug is designed to skip exon 51 of the dystrophin gene, allowing the body to make a shortened but functional form of the dystrophin protein. This shortened version may help slow muscle damage.
“This is expected to have the effect of stabilizing or improving muscle function,” noted the press release.
What is dystrophin?
Dystrophin is the protein that mutates and leads to DMD. It is found in small quantities in muscle cells, where its role is to protect the cell from daily damage.
When it mutates, it causes muscle cell degeneration and inflammation, leading to damage in the tissue of the bones, lungs and heart.
In January 2025 the FDA granted the drug Rare Pediatric Disease Designation, another program meant to support development of therapies for serious conditions affecting children. With both designations in place, the investigational treatment could move forward with additional regulatory and financial support, potentially accelerating clinical testing and review.
For patients and families living with DMD, the designation does not mean immediate access to treatment, but it signals progress toward more options in the future. Many different mutations can cause DMD; exon 51 skipping therapies target only a specific group of mutations, but they represent a meaningful step in precision medicine for Duchenne.
NS Pharma, a subsidiary of Nippon Shinyaku, is advancing the therapy’s development. The company stated that the FDA’s decision reflects the urgent need for new approaches to Duchenne and reinforces its commitment to bringing forward treatments that may improve quality of life for children affected by the disease.
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