The approval marks the first FDA-approved treatment for pulmonary arterial hypertension.
The US Food and Drug Administration (FDA) has granted approval to bosentan (Tracleer) for the treatment of pulmonary arterial hypertension (PAH) in children 3 years of age and older, to improve pulmonary vascular resistance.
The drug, produced by Actelion Pharmaceuticals, is the first FDA approved treatment for children with PAH. An orally active endothelin receptor antagonist (ERA), it was approved for a 32-mg dose with score lines to allow for physicians to vary the prescribing dose based on the patient’s weight.
It can also be dispersed in a teaspoon of water before being given orally. Bosentan was originally approved for PAH in November of 2001 for the treatment of the condition in adult patients, in doses of 62.5 mg and 125 mg.
“Actelion has focused on the needs of the PAH community since TRACLEER, our first treatment for PAH, was approved in 2001,” Gary Palmer, MD, MBA, the senior vice president of Medical at Actelion Pharmaceuticals US, Inc., said in a statement. “We’re pleased our portfolio of treatments continues to grow and pediatric PAH patients will now have an FDA-approved treatment option available.”
In its clinical trials, bosentan showed 60% effectiveness in those with World Health Organization functional class II to IV symptoms and causes of idiopathic or heritable PAH, 21% in PAH associated with connective tissue disorders, and 18% in PAH associated with congenital heart disease and left-to-right shunting.
The most common adverse events in the trial were respiratory infection (22% compare to 17% in placebo), headache (15% to 14%), edema (11% to 9%), chest pain (5% to 5%), syncope (5% to 4%), flushing (4% vs 3%), hypotension (4% vs 2%), sinusitis (4% vs 2%), arthralgia (4% vs 2%), abnormal serum aminotransferases (4% vs 2%), heart palpitations (4% vs 2%), and anemia (3% vs 0%).
The drug is expected to roll out by Q4 2017 in its 32-mg dose, while the 62.5 and 125 mg doses will remain available, according to Actelion.