Press Releases / Notices

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NEWS 2009

December 09, 2009 R & D

Nippon Shinyaku Co., Ltd. submits New Drug Application for a therapeutic agent for Myelodysplastic Syndromes (MDS) in Japan.

Nippon Shinyaku Co., Ltd. (Head office: Kyoto, Japan; President: Shigenobu Maekawa) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) of an agent for MDS, NS-17 (generic name: Azacitidine), which was licensed from a US pharmaceutical company Pharmion Corporation (now Celgene Corporation) in 2006 and has been domestically developed by Nippon Shinyaku.

The NDA submission is based on the efficacy and safety data obtained from the Japanese pivotal clinical trial conducted by Nippon Shinyaku and several clinical trials conducted internationally.  NS-17 was designated as an orphan drug in November 2008 for the treatment of patients demonstrating all FAB classifications of MDS and therefore is subject to the benefit of a priority review in Japan.  Azacitidine in an international trial of higher risk MDS patients has demonstrated a median overall survival of 24.5 months compared with 15.0 months in conventionally treated patients, a very substantial increase in median overall survival of 9.4 months (stratified log rank p=0.0001).

MDS is a group of life-threatening hematologic disorders with poor prognosis, which have a propensity to progress to acute leukemia and are generally resistant to conventional treatments.  Typical symptoms include weakness, fatigue, infections, fever, and bleeding resulting from inadequate red and white blood cell production.  Patients are often elderly and have comorbid conditions often complicated by consequences of treatment such as iron overload from frequent blood transfusions with resultant complications such as multiple organ failure.

NS-17 exerts its effects through the inhibition of DNA hypermethylation which frequently occurs in tumor cells, resulting in promoting normal development of blood cells.  Additionally, the cytotoxic effects of azacitidine cause the death of rapidly dividing cells.  Azacitidine has been already launched in more than 20 countries, and especially in the U.S., where it is registered as VIDAZA®, for the treatment of all 5 FAB subtypes of MDS and where it is used as one of the first-line therapies for MDS.  In clinical trials, Vidaza demonstrated a significant survival advantage of 9.4 months over conventional care regimens.

MDS primarily affects the elderly, and the estimated prevalence of MDS in Japan is currently about 9,000 cases.  Since a traditional treatment for MDS is mainly supportive, there has been an increasing demand for the development of novel agents to address the unmet medical needs for better therapeutic approaches for MDS.  Nippon Shinyaku believes that NS-17 and the currently existing international data will contribute to further improvement of therapy for MDS in Japan by being launched into clinical setting at an early date.