US FDA gives nod to Actelion’s PAH drug Opsumit

The US Food and Drug Administration has approved the pulmonary arterial hypertension (PAH) drug Opsumit (macitentan) from Swiss biotech firm Actelion (SIX: ATLN).

The FDA approval was based in part on data from the Phase III SERAPHIN study, a long-term study in PAH patients with predominantly WHO Functional Class II-III symptoms treated for an average of two years. Patients were treated with Opsumit monotherapy or in combination with phosphodiesterase-5 inhibitors or inhaled prostanoids. Patients had idiopathic and heritable PAH (57%), PAH caused by connective tissue disorders (31%), and PAH caused by congenital heart disease with repaired shunts (8%). Opsumit was effective in delaying disease progression, a finding that included a decline in exercise ability, worsening symptoms of PAH or need for additional PAH medication.

Jean-Paul Clozel, chief executive of Actelion, said: "Today's approval of Opsumit by the FDA is providing the PAH community with a unique treatment option, the only oral PAH medicine that has proven to delay disease progression. Over the last 14 years, Actelion has worked tirelessly to first discover and then develop Opsumit in the largest, longest and first-ever outcome study in PAH. I would like to express my gratitude to all the members of the PAH community. Without their contribution, Opsumit would not have become a reality. We will now leverage our existing PAH expertise and infrastructure to bring Opsumit to patients within the coming weeks."