Actelion announces results of the MAESTRO study with macitentan in patients with pulmonary arterial hypertension due to Eisenmenger Syndrome - Seite 3

The company will now fully analyze the data and make them available through a peer-reviewed publication.

ABOUT EISENMENGER SYNDROME
Eisenmenger Syndrome represents the most advanced form of pulmonary arterial hypertension in conjunction with congenital heart disease (PAH-CHD). The congenital heart defect causes a shunt to develop between two chambers of the heart, so an increased blood flow returns to the lungs. The blood vessels in the lung arteries become stiff and narrow, resulting in pulmonary hypertension. Eisenmenger Syndrome occurs when the pressure in the pulmonary circulation becomes so great that the direction of blood flow through the shunt reverses. It is associated with the development of chronic cyanosis and limited exercise capacity.

Patients with Down Syndrome represent between 25% and 50% of the Eisenmenger population, depending on cohort studied. To address the high unmet medical need for effective, targeted PAH therapies in this vulnerable population, Actelion extended the MAESTRO study with macitentan in Eisenmenger Syndrome patients to the Down Syndrome community. To ensure proper safeguards were established to protect the patients' rights and safety, the company worked with ethics committees, patient advocacy, support groups and patients' families.

ABOUT THE MAESTRO STUDY
MAESTRO (MAcitentan in Eisenmenger Syndrome To RestOre exercise capacity) was a Phase III multi-center, double-blind, randomized, placebo-controlled, parallel-group study to evaluate the effects of macitentan on exercise capacity in patients with Eisenmenger Syndrome. The study was started in 2013 and global enrollment was completed in August 2016 with a total of 226 patients. Patients were randomized in a 1:1 ratio, with 114 patients in the macitentan 10 mg group and 112 patients in the placebo group over a 16-week treatment period. The study was conducted in 71 centers in 26 countries. The regions included North and Latin America, Europe, and Asia-Pacific. A sub-study was conducted in 8 countries and 11 centers to evaluate the effects of macitentan on hemodynamic parameters assessed by cardiac catheterization in patients with Eisenmenger Syndrome.