The U.S. Food and Drug Administration has accepted United Therapeutics Corporation’s New Drug Application for ralinepag, an investigational oral prostacyclin receptor agonist for the treatment of pulmonary arterial hypertension (PAH).
The agency set a Prescription Drug User Fee Act target action date of June 24, 2027, according to an announcement on Monday. Ralinepag has not yet been approved for any indication and remains under investigation for PAH.
The application is supported by data from the phase 3 ADVANCE OUTCOMES trial, which enrolled 687 PAH patients predominantly pre-treated with background therapies. The study met its primary endpoint, demonstrating a 55% reduction in the risk of clinical worsening compared with placebo. Secondary measures showed reductions in N-terminal pro-B-type natriuretic peptide levels and improvements in six-minute walk distance, alongside a 47% increase in the odds of achieving clinical improvement, with statistical significance at p=0.015.
United Therapeutics said the safety profile of ralinepag was consistent with known prostacyclin-related adverse events, with no new safety signals observed. The company plans to present additional data from the ADVANCE OUTCOMES study and the open-label extension ADVANCE EXTENSION at the 2026 annual meeting of the American Thoracic Society.
PAH affects an estimated 500,000 people globally, including about 50,000 in the United States. Ralinepag is designed for once-daily oral administration, positioning it as a potential oral alternative to existing prostacyclin-based therapies for PAH management.
