News Releases & Research Results Marketing approval of NS-065/NCNP-01 (“viltolarsen”) for the treatment of Duchenne muscular dystrophy

The results of collaborative research and development conducted by the National Center of Neurology and Psychiatry (led by Director Hidehiro Mizusawa) and Nippon Shinyaku Co., Ltd. (led by President Shigenobu Maekawa).

• On Wednesday March 25, the Ministry of Health, Labour and Welfare (MHLW) approved the manufacture and sales of the National Center of Neurology and Psychiatry and Nippon Shinyaku Co., Ltd.,
• NS-065/NCNP-01 is an oligonucleotide therapeutic agent with exon-skipping effect*, which will be put into practice for the first time in Japan. This drug is expected to suppress disease progression and improve disease condition by restoring dystrophin production in patients with Duchenne muscular dystrophy.
  * A treatment method to correct the slipping of amino acid reading frames by artificially removing (skipping) one or some of the regions in a gene transcript that would be translated into a protein (exons) by using a short synthetic oligonucleotide called an antisense oligonucleotide.
• This therapeutic agent has to undergo further efficacy and safety verification through phase III trials after marketing, because it is subject to the Scheme for Conditional Early Approval besides being subject to the SAKIGAKE Review Designation System and designated as an orphan drug by the MHLW.

This R&D project was conducted with the support of the Project Promoting Clinical Trials for Development of New Drugs by AMED.