Biopharmaceutical company and leader in pulmonary arterial hypertension (PAH) therapeutics, Actelion Ltd., has just announced it has filed a New Drug Application (NDA) with the US Food and Drug Administration for innovative PAH treatment Uptravi® (selexipag) — the first oral formulation of a selective IP prostacyclin receptor agonist.
The NDA was filed along with encouraging findings from a pivotal Phase III GRIPHON study that involved 1,156 PAH patients, which showed selexipag’s ability to lower the risk of a morbidity/mortality event by 39 percent, compared to a placebo. Factors such as age, gender, WHO Functional Class, disease etiology and history of PAH therapy were considered and did not appear to significantly affect the drug’s efficacy.
The GRIPHON study treated patients for up to 4.2 years, and resulted in an overall similar tolerability profile with other available prostacyclin therapies. Noted adverse effects during treatment with selexipag were headache, diarrhea, jaw pain, nausea and vomiting, myalgia, pain in the extremities, and flushing.
Selexipag was originally discovered and developed by pharmaceutical company Nippon Shinyaku. Nippon signed a global alliance with Actelion in April 2008 to co-develop and commercialize the drug outside of Japan. It selectively targets the IP receptor, causing vasodilation and inhibiting the production of vascular smooth muscle cells. Compared to prostacyclin analogs, selexipag has been shown to maintain therapeutic effect while minimizing side effects brought about by agonism of other prostanoid receptors.
“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,” said Dr. Jean-Paul Clozel, the company’s Chief Executive Officer. “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.”
As of today, selexipag’s Marketing Authorization Application (MAA) at the European Medicines Agency (EMA) is still pending approval. Regulatory review of selexipag for PAH is also underway in New Zealand.