In an international phase III trial, ralinepag significantly reduced the risk of first clinical worsening in low-risk patients with pulmonary arterial hypertension already receiving contemporary background therapy.However, the drug was associated with more adverse event-related treatment discontinuations, the most common side effects being headache, diarrhea, nausea, and myalgia.Of note, ralinepag's comparative efficacy or tolerability against selexipag or other approved prostacyclin-pathway therapies remains unknown.
Oral ralinepag is positioned for potential FDA approval following its strong results as a treatment for pulmonary arterial hypertension (PAH) in an international phase III trial.
In ADVANCE OUTCOMES, ralinepag conferred a reduction in composite first clinical worsening events in a population of pretreated, low- or intermediate-to-low-risk patients mostly already on dual background PAH therapy (18% vs 36% for placebo; HR 0.45, 95% CI 0.33-0.62).
Ralinepag's effects, apparent by week 8 and maintained through follow-up of over 1 year, were evident for the individual outcomes of disease progression (3% vs 11%), initiation of parenteral or inhaled prostacyclin-pathway therapy (3% vs 7%), and unsatisfactory long-term clinical response (4% vs 10%). Ralinepag was also associated with favorable changes in NT-proBNP and 6-minute walk distance (6MWD) results, according to Marc Humbert, MD, PhD, of Université Paris-Saclay, INSERM, and colleagues.
