Actelion is on course to further extend its pulmonary arterial hypertension (PAH) franchise and gain European approval for Opsumit (macitentan).
The long-term PAH treatment, which was last week approved in the US, has been recommended for EU approval by EMA scientific advisers.
The ‘positive opinion’ from the CHMP puts Opsumit on course for European approval within the next three months, and Actelion will be hoping it will help shore-up revenues from its existing PAH drug Tracleer.
Tracleer (bosentan) made sales of around $1.3bn in the first nine months of this year and accounts for the bulk of the company’s revenues, but the drug is due to start losing patent protect in 2015.
Like Tracleer, Opsumit is an endothelin receptor antagonist, but its lower tendency to cause liver side effects removes the need for patient monitoring.
Opsumit was backed by the CHMP as a monotherapy, or in combination, for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients.
The positive opinion was based on the phase III SERAPHIN study, which showed Opsumit reduced the risk of a morbidity/mortality event – the primary endpoint of the study – by 45 per cent compared to placebo.
The study also found a reduction in risk of PAH-related hospitalisation or death of 50 per cent compared to placebo.
Actelion is seeking a license for the drug to treat those patients classed as WHO Functional Class II to III, that is patients for whom ordinary physical activity (Class II) or less than ordinary physical activity (Class III) can cause undue fatigue or chest pain.
Actelion’s CEO Jean-Paul Clozel said: “If approved, Opsumit could transform the lives of many people living with PAH. This positive opinion stands testament to the efforts of our development teams and our ongoing commitment to patients living with PAH.”
An incurable disorder, PAH is characterised by abnormally high blood pressure in the arteries between the heart and lungs, causing symptoms that can range from mild breathlessness and fatigue to reduced life expectancy.
Actelion has two other drugs in its PAH franchise – Ventavis (iloprost) and Veletri (epoprostenol) – but these lag behind Tracleer and brought in collective sales of just over $120m Swiss francs in the year-to-date. The company also has a further PAH treatment in its late-stage pipeline in the form of the phase III drug selexipag.