FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 to Treat Muscle Wasting Disease

The FDA has granted rare pediatric disease designation to NS-050/NCNP-03 (NS Pharma, Inc) for the treatment of Duchenne muscular dystrophy (Duchenne)—a progressive muscle wasting disease. The study authors noted that the designation could accelerate the development of the therapy to treat this disease.¹

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"The journey from first symptom to diagnosis and finally treatment can be long and challenging for patients with rare diseases and their caregivers," said Yukiteru Sugiyama, PhD, NS Pharma president, in a news release. "We are grateful for this designation which can help us accelerate the development of this therapy for Duchenne."¹

The muscle wasting disease is reported to be caused by a deficiency of the dystrophin protein, which helps keep muscle cells intact; this deficiency can result in weakness of skeletal, cardiac, and pulmonary muscles. Symptoms of Duchenne can begin in early childhood, between the ages of 2 and 3 years, primary impacting males; however, in rare cases females are also affected. Symptoms of Duchenne include muscle weakness, first occurring in the proximal muscles and then the distal limb muscles. Children impacted by the disease can experience difficulty in jumping, running, and walking, while also suffering from enlarged calves, a waddling gait, and lumbar lordosis. Due to a progressive loss of mobility, individuals with Duchenne may require the use of a wheelchair. As the disease progresses, cardiac and respiratory muscle problems could occur, beginning in the teenage years, which could result in serious, life-threatening complications.^1,2

The study authors noted that NS-050/NCNP-03 is an antisense oligonucleotide, the role of which is to skip part of the genetic information of the dystrophin gene and produce a functional dystrophin protein that has a marginally shorter chain length. The researchers provided that this is expected to have the effect of suppressing muscle function deterioration, aiding symptoms caused from Duchenne. NS-050/NCNP-03 was co-developed by National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku, according to the release.¹

While there are many types of genetic mutations that could cause Duchenne, the study authors noted that NS-050/NCNP-03 is being developed to aid individuals with confirmed gene mutations that are treatable with exon 50 skipping therapy.¹

NS Pharma noted in the release that they are working on conducting a phase 1/2 study that will evaluate the safety and efficacy of NS-050/NCNP-03 among individuals with Duchenne in the US and Japan.¹

REFERENCES

1. FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 for the Treatment of Duchenne Muscular Dystrophy. PR Newswire. News release. September 10, 2024. Accessed September 11, 2024. https://www.prnewswire.com/news-releases/fda-grants-rare-pediatric-disease-designation-to-ns-050ncnp-03-for-the-treatment-of-duchenne-muscular-dystrophy-302239736.html.

2. Duchenne Muscular Dystrophy (DMD). Muscular Dystrophy Association. News release. Accessed September 11, 2024. https://www.mda.org/disease/duchenne-muscular-dystrophy.