Actelion Ltd, a biopharmaceutical company developing innovative therapeutics for orphan diseases, submitted a Marketing Authorization Application (MAA) for their lead investigational drug selexipag (Uptravi®) to the European Medicines Agency (EMA) as a new therapeutic for pulmonary arterial hypertension.
Selexipag’s long- term efficacy and safety was previously tested in a key clinical trial, the Phase III GRIPHON study, with 1,156 patients affected by pulmonary arterial hypertension. This was the largest study to date addressing pulmonary arterial hypertension and demonstrated the efficacy of Selexipag (Uptravi®) administration in delaying the disease’s first signs of morbidity and mortality, while proving to be a safe therapeutic to be administered orally. Specifically, the trial demonstrated that Selexipag reduced the onset of disease and PAH-related death events by 39% when compared to placebo control patients during the 4-year trial period.
Pulmonary arterial hypertension is a severe disease characterized by increased blood pressure in lungs’ vasculature, due to vasoconstriction of lungs’ blood vessels. This tightening leads to symptoms of shortness of breath and dizziness, ultimately culminating in heart failure. Selexipag is a drug that is part of the prostacyclin therapies class – prostacyclin is a vasodilator substance produced by endothelial cells (these cells line the interior surface of blood vessels and lymphatic vessels) – that induce vasodilation while inhibiting the aggregation of platelets and reducing inflammation. In pulmonary arterial hypertension, as in other diseases, the prostacyclin pathway is inhibited. Selexipag is the first therapeutic that selectively targets the prostacyclin receptor (IP-receptor), to induce vasodilation and inhibit damaging signals to blood vessels (such as dampening the proliferation of vascular smooth muscle cells), improving the condition of pulmonary arterial hypertension affected patients.
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion noted, “I am delighted that we have been able to submit this application in 2014, demonstrating once again the power of our commitment to bring forward new solutions for the PAH community as rapidly as possible. With selexipag, PAH specialists may be able to target the prostacyclin pathway with an oral therapy with long-term outcome benefits. We will work closely with Health Authorities in order to be able to make this treatment available to the PAH community as quickly as possible.”
In a near future, Actelion Ltd Company will submit a Marketing Authorisation Application to the US FDA.