EU approves Opsumit for PAH-Actelion

Actelion Ltd announced that Opsumit (macitentan), a novel dual endothelin receptor antagonist (ERA), has been granted marketing authorisation on 23 December 2013, for the long-term treatment of Pulmonary Arterial Hypertension (PAH) in the EU by the European Commission. Opsumit, as monotherapy or in combination, is indicated for the long term treatment of Pulmonary Arterial Hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III.

Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.

Macitentan 10mg is also described as reducing the risk of PAH related death or hospitalization for PAH up to end of treatment (EOT) by 50% (HR 0.50; 97.5% CI: 0.34 to 0.75; logrank p < 0.0001). Macitentan 10mg improved quality of life assessed by the SF-36 questionnaire.

The EU label was based in part on data from the landmark Phase III SERAPHIN study which was published in the New England Journal of Medicine in August 2013. The SERAPHIN study demonstrated that treatment with macitentan 10 mg resulted in a 45% risk reduction (hazard ratio [HR] 0.55; 97.5% CI: 0.39 to 0.76; p < 0.0001) of the composite morbidity-mortality endpoint up to (EOT) when compared to placebo.

Opsumit was FDA approved on 18 October 2013.