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, for the application. United Therapeutics, based in Silver Spring, Maryland, and with research operations in Research Triangle Park, North Carolina, did not disclose a planned commercial launch timeline.
The NDA submission is supported by data from the Phase 3 ADVANCE OUTCOMES study, which enrolled 687 predominantly pre-treated patients with PAH. The trial met its primary endpoint, demonstrating a 55% reduction in the risk of clinical worsening compared with placebo. Secondary endpoints also showed statistically significant improvements, including a 47% higher odds of achieving clinical improvement and reductions in N-terminal pro-B-type natriuretic peptide, a biomarker associated with heart failure.
Exercise capacity, measured by six-minute walk distance, improved in the ralinepag arm relative to placebo. Subgroup analyses indicated consistent benefits across disease etiology, functional class, exercise capacity, biomarker levels, and background therapy use.
Safety findings were consistent with the known adverse event profile of prostacyclin therapies, with no new safety signals identified. Ralinepag remains investigational and has not been approved for any indication by the FDA.
PAH, a chronic and progressive disease characterized by elevated pressure in the pulmonary arteries, affects approximately 500,000 people worldwide, including about 50,000 in the United States. Study results were presented at the 2026 annual meeting of the American Thoracic Society and published in The Lancet. An open-label extension study, ADVANCE EXTENSION, is ongoing for patients who completed the ADVANCE OUTCOMES trial.
