NS Pharma announced that the FDA has granted Orphan Drug Designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne) in patients amenable to exon 51 skipping. 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-051/NCNP-04 previously received Rare Pediatric Disease Designation from the FDA in January 2025.
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac, and respiratory muscles. There are many types of genetic mutations that can cause Duchenne, and NS-051/NCNP-04 is being developed to treat patients with confirmed gene mutations amenable to exon 51 skipping therapy.
NS-051/NCNP-04 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-051/NCNP-04 promotes the skipping of exon 51 within the dystrophin gene, causing the production of a shortened dystrophin protein containing essential functional portions. This is expected to have the effect of stabilizing or improving muscle function.
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