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.