Regulatory Decision Summary for UPTRAVI

The benefits of treating patients who have idiopathic pulmonary arterial hypertension (iPAH), heritable pulmonary arterial hypertension (hPAH), PAH associated with connective tissue disorders or PAH associated with congenital heart disease with Uptravi whether combined or as monotherapy clearly outweigh the risks. Uptravi is seen as having a favorable benefit/risk profile. The pivotal trial showed both clinically and statistical significant improvements in morbidity events. The delay in hospitalization for worsening of PAH and the delay in disease progression resulting from the use of selexipag are considered as being of benefit to the patient, especially in terms of improved quality of life. With respect to safety and risk, considering the very serious nature of the underlying disease, no significant or unmanageable safety concerns were noted to arise in the course of either the pivotal study or other studies.

The principle uncertainty associated with the use of Uptravi relates to its lack of effect on mortality; use of the drug had no effect on mortality in the pivotal study. The benefit obtained from its use is primarily in that it delays time to hospitalization for PAH and reduces to some extent the progression of the disease. It does not extend the life span of the PAH patients on whom it is used.

For more information on Health Canadas decision, please view the Summary Basis of Decision.