FDA approves PAH orphan drug treatment

SILVER SPRING, Md. — The Food and Drug Administration on Monday approved Actelion Pharmaceutivals' Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension, a chronic rare lung disease that can lead to death or the need for lung transplantation.

PAH is high blood pressure that occurs in the arteries that connect the heart to the lungs, and causes the right side of the heart to work harder than normal, which can lead to limitations on exercise ability and shortness of breath, among other more serious complications.

Uptravi belongs to a class of drugs called oral IP prostacyclin receptor agonists. The drug acts by relaxing muscles in the walls of blood vessels to dilate (open) blood vessels and decrease the elevated pressure in the vessels supplying blood to the lungs.

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