Actelion's Opsumit (macitentan) has won approval in the European Union as a treatment for pulmonary arterial hypertension (PAH) in adults.
A follow-up to its hugely successful Tracleer (bosentan), which goes off-patent in 2015, the drug is a novel dual endothelin receptor antagonist indicated as a monotherapy or in combination for the long-term treatment of PAH, a condition characterised by abnormally high blood pressure in the arteries between the heart and lungs.
While advances in research have shifted the prognosis and treatment of PAH to that of a long-term condition, it does remain a progressive disease with high morbidity and mortality, the firm noted, stressing the need for new an effective therapies.
“For many years, the PAH medical community has been asking for a drug with a demonstrated benefit in reducing morbidity-mortality events, and for the first time, with macitentan (Opsumit), we have this," said Luke Howard, Consultant Respiratory Physician, Hammersmith Hospital, Imperial College Healthcare NHS Trust, London.
"This new treatment provides meaningful benefits for patients, importantly both in those who are treatment-naïve, as well as those already on PAH-specific therapies (mainly PDE5 inhibitors).” he added.
Approval was partly based on data from the Phase III SERAPHIN trial, in which patients taking the drug had a 45% risk reduction of the composite morbidity-mortality endpoint compared to placebo.
The risk of PAH-related death or hospitalisation was also slashed, by 50%, during the trial, and the drug was found to be generally well-tolerated, with the most commonly reported adverse drug reactions nasopharyngitis (14.0%), headache (13.6%) and anaemia (13.2%), according to Actelion.
EU approval of Opsumit follows the thumbs up from the European Medicines Agency's Committee for Human Medicinal Products (CHMP) and a US green light in October.