Tracleer Becomes First FDA-approved Therapy in U.S. for Children with Pulmonary Hypertension

Alice Melão, MSc avatar

by Alice Melão, MSc |

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Tracleer (bosentan) has become the first therapy to obtain U.S. regulatory approval for treating pulmonary hypertension in children.

The Food and Drug Administration authorized it for children aged 3 and older who have idiopathic or congenital pulmonary arterial hypertension (PAH).

Actelion Pharmaceuticals, the company that holds the marketing license for Tracleer, expects to make the  children’s version of it available by the fourth quarter of 2017. It will be a 32-mg oral-suspension tablet.

“Actelion has focused on the needs of the PAH community since Tracleer, our first treatment for PAH, was approved in 2001,” Dr. Gary Palmer, senior vice president of Actelion Pharmaceuticals US, said in a press release. “We’re pleased our portfolio of treatments continues to grow, and pediatric PAH patients will now have an FDA-approved treatment option available.”

Tracleer is an endothelin receptor antagonist that works by reducing the amount of a substance called endothelin in the blood. Endothelin causes blood vessels in the lungs to narrow, increasing blood pressure in the pulmonary arteries.

The FDA had already approved it for adults with pulmonary hypertension that fell into the WHO group 1 category, which covers patients with the least severe symptoms. Tracleer is designed to improve patients’ ability to exercise and slow the progression of their disease.

Parents can put the children’s formulation into water to make it easier for them to take. Doctors need to take patients’ weight into consideration when prescribing a dose. Marks on the tablets that indicate doses below 32 mg will help them do that.

Actelion makes the adult version of Tracleer in 62.5-mg and 125-mg doses.

The European Union approved Tracleer as a treatment for children with PAH in 2009. The approval covers children aged 1 and older.

Several clinical trials, including the FUTURE-1 (NCT00319267) and FUTURE-2 (NCT00319020) studies, have shown that Tracleer is safe in children and that they tolerate it well. In terms of effectiveness, the trials showed that the drug had the potential to keep children’s disease symptoms stable.

The trials have also demonstrated that Tracleer’s safety and tolerability profiles in children were similar to those reported in placebo-controlled clinical trials of adults with PAH.