Actelion has submitted its new treatment for pulmonary arterial hypertension (PAH) to the U.S. Food and Drug Administration (FDA). The company is applying for a New Drug Application (NDA) for selexipag (Uptravi), after having also submitted the drug to the European Medicines Agency (EMA).
Actelion is expecting to offer selexipag as a treatment for patients who suffer from PAH, which will make it the first selective oral IP prostacyclin receptor agonist for the disease. The drug, which was originally developed by the Kyoto, Japan-based research company Nippon Shinyaku, has been evaluated in a phase III pivotal study called GRIPHON, whose encouraging results supported Actelion’s recent submission efforts.
GRIPHON included 1,156 PAH patients and demonstrated that selexipag was able to diminish the morbidity/mortality risk in the disease by 39%, when compared to placebo. In addition, the revealed efficacy was consistent in all key subgroups of the study, including age, gender, WHO Functional Class, PAH etiology and background PAH therapy. The scientists demonstrated that selexipag’s tolerability profile was similar to prostacyclin therapies, and adverse reactions occurred in less than 5% of the cases and included headache, diarrhea, jaw pain, nausea, myalgia, vomiting, pain in extremity, and flushing.
“Successfully submitting the selexipag NDA dossier to the FDA is another great milestone for us at Actelion, and a great way to mark the end of a remarkable year for us all,” stated the Chief Executive Officer of Actelion, Jean-Paul Clozel, M.D. “With the submission process underway for selexipag with both the FDA and EMA, I am looking forward to 2015 and to continuing to work with health authorities to move forward with these submissions in order to make selexipag available to the PAH community at the earliest opportunity.”
Actelion submitted a Marketing Authorisation Application (MAA) at the beginning of December to the EMA for selexipag, and the application is currently pending validation. In addition, there is also an ongoing regulatory review being conducted for the drug in New Zealand. According to the licensing agreement signed by Actelion and Nippon Shinyaky in April 2008, Actelion is responsible for the global development and commercialization of selexipag outside Japan, while Nippon is developing and commercializing it in Japan.