The US Food and Drug Administration (FDA) made August a big month for Actelion Pharmaceuticals. On August 10 the company announced it received FDA approval for a new, stronger concentration of the pulmonary arterial hypertension (PAH) treatment, Ventavis (Iloprost). On the same day, the FDA expanded the approval of Tracleer (bosentan) in Functional Class II PAH, allowing patients with earlier stages of PAH to benefit from the oral treatment.


The FDA approved Actelion’s supplemental New Drug Application (sNDA) for Tracleer to treat patients with mildly symptomatic World Health Organization (WHO) Functional Class II (FC II) PAH. The FDA also approved Actelion’s Risk Evaluation and Mitigation Strategy (REMS) for Tracleer.

“Physicians in the United States of America who treat PAH patients now have access to Tracleer as an approved therapy for patients not only in advanced, but also in the early stages of this rapidly progressing and life-threatening disease. This label extension is based on EARLY, the only randomized, double blind, placebo-controlled study in this mildly symptomatic patient population. The EARLY results demonstrate that Tracleer significantly reduces risk of clinical worsening in early-stage patients, thereby slowing down disease progression,” said Jean-Paul Clozel, MD, chief executive officer of Actelion, in an announcement.

“The FDA’s approval of Tracleer for use in patients with early-stage PAH provides the opportunity to improve clinical outcomes. Tracleer is the only PAH medication that has consistently shown significantly reduced risk of clinical worsening in PAH patients in three separate Phase III studies,” said Kirk Taylor, MD, senior vice president, US medical group at Actelion Pharmaceuticals US Inc.

According to Actelion, Tracleer is an oral dual endothelin receptor antagonist approved for the treatment of PAH FC II, III and IV in the United States and for the treatment of PAH FC II and III in the European Union. The company is working with authorities on a worldwide basis to expand the label for Tracleer to include patients with FC II PAH.


Ventavis received FDA approval for increased 20 mcg/ml strength formulation as a therapy for PAH WHO Group 1 in patients with New York Heart Association Class III and IV symptoms. According to Actelion, this new increased strength formulation will deliver the same dose in half the volume, which is expected to reduce inhalation time and further support patient compliance.

The approval was based on the submission of technical data showing that the new formulation did not alter the functional characteristics of the delivery system or the emitted dose to the patient.

“We have now been successful with this new strengthened formulation offering additional convenience for US patients using Ventavis. We will continue our commitment to Ventavis as well as our portfolio of PAH programs in order to achieve further improvements for the PAH community,” said Clozel in an announcement about the approval.