Japan Approves Uptravi for Pediatric PAH

PAH is a disease with a poor prognosis characterized by abnormally high blood pressure in the pulmonary artery. It is classified into idiopathic PAH, heritable PAH and PAH associated with various diseases such as connective tissue disease and congenital heart disease. Because the disease pathology of PAH is similar between pediatric and adult patients, combination therapy of prostacyclin pathway drug, endothelin receptor antagonist and phosphodiesterase-5 inhibitor, is also recommended for pediatric PAH patients. However, treatment options for pediatric PAH are limited in Japan. Particularly, in the case of prostacyclin pathway drug, only an injectable formulation requiring continuous intravenous infusion is available. Therefore, there has been an unmet need for oral formulations of prostacyclin pathway drug. 

Uptravi is an oral prostacyclin receptor (IP receptor) agonist with high selectivity for the IP receptor among prostacyclin pathway drugs. Uptravi is believed to reduce pulmonary arterial pressure by binding to the IP receptors on vascular smooth muscle cells and increasing cAMP production, thereby leading to vasodilation and inhibition of vascular smooth muscle proliferation.on Uptravi therapy, as uninterrupted treatment is considered key for individuals with PAH. Uptravi tablets were first approved by the FDA in 2015 to delay disease progression and reduce the risk of hospitalization for PAH.

The optimal dose of Uptrav for each patient is determined by dose titration. The new 0.05 mg tablet allows for finer dosing for pediatric patients, but some patients may need to take a larger number of tablets at one time. To support patents and healthcare professionals who take and dispense the drug,the company e developed a new pill case especially for the pediatric formulation.