FDA Grants Rare Pediatric Disease 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 Rare Pediatric Disease Designation to NS-065/ NCNP -01※1 which is being developed for the treatment of Duchenne Muscular Dystrophy (DMD) .
The Rare Pediatric Disease is defined as a disease that affects fewer than 200,000 persons in the US, primarily aged from birth to 18 years. Under this designation, when an approval is granted for NS-065/NCNP-01, Nippon Shinyaku may be eligible to obtain priority review for a subsequent marketing application for a different product. NS-065/NCNP-01 was previously granted Fast Track Designation in October, 2016 and Orphan Drug Designation in January this year 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 started in March 2016 in the US. "SAKIGAKE designation"※2 of the Ministry of Health, Labor and Welfare was registered, and then a phase 1/2 clinical study started in January 2016 in Japan.
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.
※1 NS-065/NCNP-01 is a morpholino antisense oligonucleotide, which is expected to improve muscle function for DMD. It was co-discovered by Nippon Shinyaku and National Center of Neurology and Psychiatry (NCNP: Kodaira City, Tokyo; President, Hidehiro Mizusawa, Director General, National Institute of Neuroscience, Shin'ichi Takeda) and is a drug candidate which is expected to be an important option for DMD amenable to exon 53 skipping.
※2 "SAKIGAKE designation (Japanese version of Breakthrough Therapy Designation) " is to promote R&D in Japan aiming at early practical application for world's first, domestically-produced and innovative pharmaceutical products for serious and life-threatening diseases. This system is aiming to shorten reviewing time for approval, facilitating a prioritized consultation and review for regulatory approval.