New PAH Drug from Actelion Approved by FDA

According to a recent announcement, the United States Food and Drug Administration (FDA) approved Actelion Pharmaceuticals’ Uptravi (selexipag) tablets for the treatment of adult patients with pulmonary arterial hypertension (PAH) to delay disease progression and reduce the risk of hospitalization, under orphan drug designation.

PAH is a life-threatening disease characterized by high blood pressure and abnormal constriction of the pulmonary artery. The condition causes the heart to work faster and elevates blood pressure in arteries within the lungs, potentially causing fatal heart failure and other, less serious, complications and disturbances in quality of life.

Uptravi is a highly selective oral prostacyclin receptor (IP) agonist. Activation of the IP receptor induces vasodilation, which in turn relaxes muscles in the blood vessels’ walls, decreases blood pressure in the vessels supplying blood to the lungs, and inhibits growth and proliferation of vascular smooth muscle cells.

The efficacy and safety of the drug were determined in a Phase III clinical trial, the GRIPHON study, which included 1,156 PAH patients between the ages of 18 and 75, representing the largest trial conducted in PAH. Uptravi was found to significantly decrease the risk of morbidity/morbility events by 40% when compared to the placebo control group, at different dosing scales. Observed adverse events included jaw pain, headache, diarrhea, and nausea. The results were presented in October 2015, at the American College of Chest Physicians’ CHEST Congress in Montréal, Canada.

Orphan drug status is given to drug candidates aiming to treat rare diseases or conditions that affect less than 200,000 individuals, a criteria met by PAH. The designation grants several development advantages for the drug and its specific indication, such as tax credits for qualified clinical testing, exemptions from certain costs under the Prescription Drug User Fee Act (PDUFA), and seven-year marketing exclusivity after the drug’s approval.

Ellis Unger, MD, director of the Office of Drug Evaluation I in the FDA’s Center for Drug Evaluation and Research commented on Uptravi’s approval, “Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension. The FDA supports continued efforts to provide new treatment options for rare diseases.”

Actelion Pharmaceuticals also submitted Uptravi to the European Medicines Agency (EMA) and the drug is currently under review.