EC approves Pfizer's Revatio for PAH in children; Savient files Krystexxa with EMA

US drugs behemoth Pfizer (NYSE: PFE) yesterday revealed that Revatio (sildenafil citrate, which is also the active ingredient of its erectile dysfunction drug Viagra) has been approved by the European Commission for the treatment of pediatric patients aged one to 17 years old with pulmonary arterial hypertension.

“Pulmonary arterial hypertension is a rare, devastating disease that can affect children," said Cara Cassino, vice president, Pfizer Medicines Development Group, adding: “With the approval of Revatio, these young patients now have an important treatment option that may help manage their condition. This approval is another example of our ongoing commitment to rare diseases.”

The approval was based on results of a dose-ranging Phase III study that evaluated the efficacy and safety of Revatio versus placebo in 234 pediatric patients with primary pulmonary hypertension or pulmonary hypertension associated with congenital heart disease. The primary endpoint was improvement from baseline in exercise capacity as assessed by change in peak volume of oxygen consumption (peak VO2) following 16 weeks of treatment. In children who were deemed developmentally unable to perform the test due to young age or the presence of other conditions, efficacy was assessed using secondary endpoints, including hemodynamics and change in WHO functional class.