Nippon Shinyaku Co., Ltd. has received rare pediatric disease designation for NS-050/NCNP-03, being developed for the treatment of Duchenne muscular dystrophy (Duchenne). The FDA's rare pediatric disease designation is for treatments of serious or life-threatening diseases in children where there are fewer than 200,000 patients in the United States.
Duchenne is a muscle wasting disease that occurs primarily in males and is caused by a deficiency of the dystrophin protein. There are many types of genetic mutations that can cause Duchenne, and NS-050/NCNP-03, an antisense oligonucleotide, is being developed to treat patients with confirmed gene mutations amenable to exon 50 skipping therapy. 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.
It is not available yet, the company is preparing clinical trials of NS-050/NCNP-03 in patients with Duchenne in Japan and the United States.
