Selexipag, Originally Discovered and Synthesized by Nippon Shinyaku, Granted Orphan Drug Designation for the Indication of Chronic Thromboembolic Pulmonary Hypertension.
Nippon Shinyaku Co., Ltd. today announced that NS-304 (generic name: Selexipag) was granted orphan drug designation by the Ministry of Health, Labor and Welfare (MHLW) for the indication of chronic thromboembolic pulmonary hypertension (CTEPH) as of June 20.
Orphan drug is defined as a medication of which medical needs are particularly high for a disease of which patients are less than 50,000 in Japan. CTEPH is a disease caused by reduced blood flow due to blockage of organized thrombus (embolization) in the pulmonary vessel, and it is designated as an intractable disease and it is reported that there are approximately 2,500 patients in Japan.
Selexipag is an orally available, long-acting prostacyclin receptor (IP receptor) agonist which selectively targets the IP receptor and shows vasodilation effect and improves pulmonary hemodynamic status.
In September 2014, Selexipag was granted orphan drug designation by the MHLW for the indication of pulmonary arterial hypertension (PAH). It has been jointly developed with Actelion Pharmaceuticals Japan Ltd in Japan, and an NDA was submittted in January this year. It has been developed by our licensee Actelion (Switzerland) overseas, and the product was launched in the United States in January this year, approved in Europe in May, and launched in Germany in June.
Additionally, as for the CTEPH indication for Selexipag, we initiated a Phase III clinical study in June this year in Japan.
With the granted orphan drug designation for the indication of CTEPH for Selexipag, we can expect that the time required to obtain manufacturing and marketing approval will be shortened by several months.
Nippon Shinyaku will continue to make further efforts going forward, so that we can deliver the medication to patients suffering from CTEPH as soon as possible.