Orphan Drug Designation of Pulmonary Arterial Hypertension Drug Selexipag
Nippon Shinyaku, Co., Ltd announced that the Ministry of Health, Labor and Welfare (MHLW) has granted orphan drug designation, as of September 17th, to the investigational drug NS-304 (Selexipag) as an agent for pulmonary arterial hypertension (PAH).
PAH is a life-threatening disease in which blood pressure in the arteries between the heart and lungs becomes abnormally elevated, and can develop without known reasons or associated with certain diseases such as connective tissue diseases and congenital heart diseases.
An orphan drug designation is granted by Minister of Health, Labor and Welfare to drugs with high medical needs which are intended to treat a disease with less than 50,000 patients, and provides several supports including shortened time period for review of application for marketing authorization.
Selexipag is an orally available, long acting prostaglandin I2 (PGI2) receptor agonist.
In counties except for Japan, a global Phase 3 study (GRIPHON) for PAH has been completed by our licensee Actelion Pharmaceuticals Ltd.(Allchwil, Switzerland). Actelion is preparing for regulatory filing with the Health Authorities.
In Japan, Nippon Shinyaku and Actelion Pharmaceuticals Japan are jointly conducting Phase 2 studies in patients with PAH and Chronic Thromboembolic Pulmonary Hypertension.
Nippon Shinyaku is determined to continue our best efforts in order to deliver selexipag to patients suffering from PAH as soon as possible.