The FDA has approved Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation. The drug is manufactured by Actelion Pharmaceuticals.
Uptravi is an oral IP prostacyclin receptor agonist that relaxes muscles in the walls of blood vessels in order to dilate them and decrease the elevated pressure in the vessels supplying blood to the lungs.
The safety and efficacy of the drug, which was granted orphan drug designation by the FDA, were established in a long-term clinical trial of 1,156 participants with PAH, according to the agency. Uptravi was shown to be effective in reducing hospitalization for PAH and reducing the risks of disease progression compared to placebo. Participants were exposed to Uptravi in this trial for a median duration of 1.4 years. Common side effects included headache, diarrhea, jaw pain, nausea, muscle pain (myalgia), vomiting, pain in an extremity, and flushing.
“Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension,” said Ellis Unger, MD, director of the Office of Drug Evaluation I in the FDA’s Center for Drug Evaluation and Research. “The FDA supports continued efforts to provide new treatment options for rare diseases.”