The U.S. Food and Drug Administration granted rare pediatric disease designation to NS-050/NCNP-03, a treatment developed for Duchenne muscular dystrophy (Duchenne), as announced by NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd. The designation—given to drugs developed for serious or life-threatening diseases affecting fewer than 200,000 patients younger than 18 in the United States—was designed to accelerate treatment development.

Duchenne muscular dystrophy, a progressive condition, leads to muscle weakness—primarily affecting skeletal, cardiac, and pulmonary muscles—due to dystrophin protein deficiency. NS-050/NCNP-03, an antisense oligonucleotide, targets patients with genetic mutations amenable to exon 50 skipping therapy. The agent functions by skipping part of the dystrophin gene to produce a shorter yet functional dystrophin protein, with the aim of slowing muscle deterioration.

NS Pharma, in collaboration with the National Center of Neurology and Psychiatry, co-discovered NS-050/NCNP-03. Phase I/II clinical trials have been planned to assess the treatment’s safety and efficacy in patients with Duchenne in Japan and the United States.