PAH patients in England denied access to Actelion’s Uptravi on NHS

Actelion’s Uptravi will not be funded on the NHS for patients with Pulmonary Arterial Hypertension (PAH), after the drug failed to make it into NHS England’s latest round of specialised commissioning approvals.

The drugmaker has slammed the decision, which it argues will delay access to Uptravi (selexipag) as a combination treatment for PAH in adult patients with one of the more severe forms of the disease who are insufficiently controlled on oral treatment.

"This ruling will create a disparity in access to selexipag throughout the UK, with patients in Scotland and Wales already having access," it stressed.

The Scottish Medicines Consortium backed NHS funding for the drug back in May, but restricted its use to combination therapy in a sub-population of patients with PAH specifically those in WHO FC III who are insufficiently controlled with an ERA and a PDE-5 inhibitor and who would be considered for treatment with inhaled iloprost.

This was followed by a green light from the All Wales Medicines Strategy Group (AWMSG) in June, which recommended the drug as part of a triple combination therapy for the treatment of the condition, in adults with one of the more severe forms of the disease who are insufficiently controlled on oral treatment with two other classes of PAH medicines.

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 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).

"Selexipag is a step-change in the treatment of these patients as it is the first oral medicine targeting the important prostacyclin pathway, which offers hope for those who have had insufficient benefit from existing oral treatments. It is therefore extremely disappointing that PH clinicians will not be able to offer this treatment to patients," said Dr John Wort, clinical lead for pulmonary hypertension at the Royal Brompton & Harefield NHS Trust.

Also expressing disappointment at NHS England’s decision, Dr Iain Armstrong, chair, Pulmonary Hypertension Association UK called for an "urgent review" on decisions regarding new therapies available for PH currently blocked or deferred by national funders.

"As a PH community we don’t ask for much; we just want the same equality and access to treatment as other disease areas like cancer. We are extremely disappointed by this decision and as a dynamic patient organisation we will continue to fight for equality and equity of access to service provision in the UK."

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