RT Journal Article SR Electronic T1 Selexipag: an oral, selective prostacyclin receptor agonist for the treatment of pulmonary arterial hypertension JF European Respiratory Journal JO Eur Respir J FD European Respiratory Society SP 874 OP 880 DO 10.1183/09031936.00137511 VO 40 IS 4 A1 Simonneau, Gérald A1 Torbicki, Adam A1 Hoeper, Marius M. A1 Delcroix, Marion A1 Karlócai, Kristóf A1 Galiè, Nazzareno A1 Degano, Bruno A1 Bonderman, Diana A1 Kurzyna, Marcin A1 Efficace, Michela A1 Giorgino, Ruben A1 Lang, Irene M. YR 2012 UL http://erj.ersjournals.com/content/40/4/874.abstract AB In this phase 2 proof-of-concept study we examined the safety and efficacy of selexipag, an orally available, selective prostacyclin receptor (IP receptor) agonist, as a treatment for pulmonary arterial hypertension (PAH). 43 adult patients with symptomatic PAH (receiving stable endothelin receptor antagonist and/or a phosphodiesterase type-5 inhibitor therapy) were randomised three to one to receive either selexipag or placebo. Dosage was up-titrated in 200-μg increments from 200 μg twice daily on day 1 to the maximum tolerated dose by day 35 (maximum allowed dose of 800 μg twice daily). Change in pulmonary vascular resistance at week 17 expressed as a percentage of the baseline value was the primary efficacy end-point, and was analysed in the per protocol set first and then in the all-treated set to assess robustness of results. A statistically significant 30.3% reduction in geometric mean pulmonary vascular resistance was observed after 17 weeks' treatment with selexipag compared with placebo (95% confidence limits -44.7– -12.2; p=0.0045, Wilcoxon rank sum test). This was supported by a similar result from the all-treated set. Selexipag was well tolerated with a safety profile in line with the expected pharmacological effect. Our results encourage the further investigation of selexipag for the treatment of PAH.