First pediatric PAH therapy approved in EU

Actelion’s pediatric dispersible formulation of Tracleer (bosentan) for the treatment of pulmonary arterial hypertension (PAH) has been approved in the EU.

The approval makes Tracleer the only PAH therapy with an approved pediatric formulation for treating children from two years of age.

The therapy has also recently been proven to be a cost-effective treatment for adult PAH patients.

PAH is a severe condition in children with an estimated median survival of 10 months after diagnosis if left untreated. The new dispersible tablet formulation of bosentan allows a more flexible dosing regimen according to low body weight.

Professor Maurice Beghetti, Head of the Pediatric Cardiology Unit at Hôpital des Enfants, Geneva, commented: "Ensuring correct dosing for children is a challenge we face across all diseases but particularly in orphan diseases that affect children. This pediatric formulation for Tracleer is a large step in the right direction towards developing treatment with the needs of children specifically in mind."

Tracleer is currently approved in Europe for the treatment of PAH; in WHO Functional Class III to improve exercise capacity and symptoms and in WHO Functional Class II where some improvements have also been shown.

Recent research using a cost utility model has suggested that Tracleer is a cost effective first-line treatment option for patients classified as WHO functional class III.

The research was published online in Value in Health, the journal of the International Society for Pharmacoeconomics and Outcomes Research, and simulated the treatment of 10,000 hypothetical patients in two treatment groups: Tracleer plus palliative care and palliative care only.

Dr Matt Stevenson, lead researcher, University of Sheffield, commented: “Long-term follow-up data from randomised controlled trials show that bosentan significantly delays progression to more severe states compared with no active intervention. This results in both potential cost-savings due to a reduction in the duration of relatively expensive epoprostenol treatment and in an improvement in the quality of life of patients.”