Actelion Pharmaceutical UK’s new endothelin receptor antagonist Opsumit (macitentan) is now available through NHS Scotland as monotherapy or in combination, for the long-term treatment of pulmonary arterial hypertension (PAH), in adult patients of WHO Functional Class (FC) II to III, following positive advice from the Scottish Medicines Consortium.

Scottish Pulmonary Vascular Unit respiratory consultant Martin Johnson said macitentan (Opsumit) provides a PAH therapy option for the first time with evidence of improvement in a combined morbidity and mortality endpoint, both as a monotherapy and in combination.

"The SMC positive advice means that this new treatment, which has demonstrated meaningful, long-term clinical benefits, is now available to PAH patients through NHS Scotland," Johnson said.

Opsumit secured EU marketing authorisation in December 2013 following completion of the landmark SERAPHIN trial, which is claimed to be the longest and largest clinical trial to be carried out so far in patients with PAH.

The drug’s efficacy has been demonstrated in a PAH population including idiopathic and inherited PAH, PAH associated with connective tissue disorders as well as with corrected simple congenital heart disease.

"This new treatment, has demonstrated meaningful, long-term clinical benefits."

In addition, Opsumit also showed efficacy in treatment naïve patients and those already receiving PAH-specific background therapies, such as phosphodiesterase type 5 inhibitors (PDE 5i).

In Scotland, currently there are an estimated 250 people on disease-targeted drug therapies for their PAH, a severe, life-threatening disorder characterised by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual.

Actelion Pharmaceuticals UK general manager Robin Bhattacherjee said Scotland is one of the first countries in Europe to make Opsumit available to prescribe.

"This milestone further demonstrates Actelion’s ongoing commitment to the discovery, development and commercialisation of innovative treatments, which offer real benefits for patients," Bhattacherjee added.

Most commonly reported adverse drug reactions with Opsumit are nasopharyngitis (14.0%), headache (13.6%) and anaemia (13.2%) and the majority of adverse reactions reported were mild to moderate in intensity.

Image: PAH is characterised by abnormally high blood pressure. Photo: courtesy of Patho.