The U.S. Food and Drug Administration (FDA) granted approval to Actelion’s Tracleer (bosentan) tablet for use in pediatric patients ages 3 years and older with idiopathic or congenital pulmonary arterial hypertension (PAH).
The drug, which is the country’s first FDA-approved therapy for pediatric PAH patients, comes in a new 32 mg tablet and is to be taken orally. It is intended to improve pulmonary vascular resistance (PVR), resulting in improved ability to exercise.
In PAH, there are issues with blood flow through the lungs, which can force an affected person’s heart to work harder. Symptoms of the life-threatening condition are much like those observed with other, more common heart and lung conditions, like abnormally high blood pressure in the pulmonary artery. As a result, the disease may not be properly diagnosed until symptoms progress and become severe.
If not properly treated, arteries in a patient’s lungs can become thick and stiff. PAH typically affects people in their late 40’s and early 50’s, and almost four times as many women are diagnosed than men. Per the American Thoracic Society
, there are an estimated 10-20,000 PAH patients living in the United States.
Tracleer is an oral endothelin receptor antagonist (ERA), which is effective because of the high endothelin levels present in PAH patients and constrict blood vessels.
"Actelion has focused on the needs of the PAH community since Traceleer, our first treatment for PAH, was approved in 2001," said Gary Palmer, M.D., MBA, senior vice president, Medical, Actelion Pharmaceuticals US, Inc. 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 has been commercially available in the U.S. since November 2001, and Actelion expects to make the 32-mg dosage option of the drug available by the fourth quarter of 2017. Until then, the 62.5 and 125-mg dosages for adult use will remain available.
The most common adverse events (AE) in patients taking Tracleer are respiratory tract infection, headache, chest pain, syncope, flushing, hypotension, sinusitis, arthralgia, serum aminotransferases abnormal, palpitations, and anemia. The drug comes with a boxed warning that makes note of the risk of hepatotoxicity and birth defects.
For more information on FDA applications, designations and approvals, follow Rare Disease Report
. To get breaking FDA news sent straight to your inbox, sign up for the RDR e-newsletter