FDA Grants Fast Track Designation to NS-065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy
Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the Food and Drug Administration (FDA) has granted Fast Track Designation to NS-065/NCNP-01 for the treatment of Duchenne Muscular Dystrophy (DMD). Fast Track is a process designed to expedite the review of drugs which will be highly effective to treat intractable diseases. NS-065/NCNP-01 is the first antisense oligonucleotide discovered in Japan to be granted Fast Track Designation from the FDA.
DMD is an inherited muscle disorder with the highest incidence that is limited to male children. It causes a severe loss of muscle power due to a deficiency of normal dystrophin, a protein involved in constructing the framework of muscle cells. This deficiency arises from mutation of the dystrophin gene. Because there is no effective treatment for DMD other than steroids, the development of an effective new treatment is desired.
NS-065/NCNP-01 is a morpholino antisense oligonucleotide, which is expected to improve muscle function by skipping a part of genetic information of the dystrophin gene. A phase 2 clinical study in the US and a phase 1/2 study in Japan commenced in March and January this year, respectively. NS-065/NCNP-01 was registered as "pioneer review designation system" of the Ministry of Health, Labour and Welfare in Japan in October 2015. The clinical study in the US is being conducted by NS Pharma, Inc. (Headquarters, New Jersey, US; President, Masato Matsuoka) which is a US subsidiary of Nippon Shinyaku.
Nippon Shinyaku has been working actively having a sense of mission to develop agents for the treatment of intractable and rare diseases, with a view to launching products for patients as soon as possible. With the Fast Track Designation, we hope to promptly provide NS-065/NCNP-01 to DMD patients.