NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the FDA granted rare pediatric disease designation to NS-050/NCNP-03 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne). The FDA's rare pediatric disease designation is granted for treatments of serious or life-threatening diseases that affect children under the age of 18 and fewer than 200,000 patients in the United States.
"The journey from first symptom to diagnosis and finally treatment can be long and challenging for patients with rare diseases and their caregivers," said NS Pharma President Yukiteru Sugiyama, Ph.D. "We are grateful for this designation which can help us accelerate the development of this therapy for Duchenne."
Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac and pulmonary muscles. There are many types of genetic mutations that can cause Duchenne, and NS-050/NCNP-03 is being developed to treat patients with confirmed gene mutations amenable to exon 50 skipping therapy.
NS-050/NCNP-03 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-050/NCNP-03 skips part of the genetic information of the dystrophin gene and produces a functional dystrophin protein with a slightly shorter chain length, which is expected to have the effect of suppressing muscle function deterioration.
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