Actelion Pharmaceuticals Trading (Shanghai) Co. Ltd.

Pulmonary hypertension is a progressive, debilitating disease caused by increased pulmonary vascular tone and vascular remodeling. Recent research evidence suggests that endothelin receptor antagonists (ERAs) are effective drugs for the treatment of PAH. Macitentan is a second-generation ERA that exerts pulmonary vasodilator effects without causing the significant side effects observed with previous ERAs. In 2017, macitentan was approved by the National Medical Products Administration for the treatment of PAH. The purpose of this study was to evaluate the effectiveness and safety of macitentan in Chinese adult patients with pulmonary hypertension.

The primary objective of this study was to evaluate the effect of selexipag compared with placebo on PVR at week 20 in subjects with inoperable CTEPH (i.e., technically inoperable) and persistent/recurrent CTEPH after surgical (PEA) and/or interventional (BPA) treatment.

Primary objective: The primary objective of this study was to evaluate the efficacy of miglustat on the rate of disease progression and disease stabilization by measuring the deterioration of saccadic eye movements in NPC subjects. Secondary objectives: (1) to evaluate the effects of miglustat on other aspects of NPC disease control; (2) to evaluate the safety and tolerability of miglustat in Chinese NPC subjects.