Some patients with pulmonary arterial hypertension (PAH) are now able to access Actelion’s selexipag on the NHS in Wales after a green light from the All Wales Medicines Strategy Group (AWMSG).

PAH is a progressive disease with high rates of morbidity and mortality affecting around 6,000-7,000 people in the UK. It is characterised by abnormally high blood pressure in the arteries between the heart and lungs, causing symptoms such as such as breathlessness, fatigue, weakness and angina.

While options are available to address these symptoms there is currently no treatment that slows progression or cures the disease, and fewer than 40 percent of patients live beyond five years of diagnosis.

Uptravi's (selexipag) European approval in May 2016 came on the back of data from the Phase III GRIPHON study showing that the drug, a selective IP prostacyclin receptor agonist, delayed the time to a first morbidity or mortality event compared to placebo.

In the trial, exposure to the drug was up to 4.2 years with a median duration of exposure of 1.4 years; the risk of this primary composite end-point was reduced by 40 percent with Uptravi compared to placebo (a relative risk reduction of 16 percent).

In its final appraisal, the AWMSG recommended use of the drug as an option for restricted use within NHS Wales as a triple combination therapy for the treatment of the condition.

Specifically, it will be funded in adults with one of the more severe forms of the disease (World Health Organisation functional class III) who are insufficiently controlled on oral treatment with two other classes of PAH medicines.

The decision represents “another step-change in the treatment of PAH,” said Dr Luke Howard, consultant pulmonologist, London Imperial College Healthcare NHS Trust.

“Access to selexipag …marks the first medicine of its kind in a tablet format to work on the prostacyclin pathway, a major disease pathway involved in the development of PAH. It is therefore great news that Wales is one of the nations in the UK leading the way in access to this innovative medicine.”

The drug is already available to patients with the condition on the NHS in Scotland.