Disappointment for Scottish patients with PAH as SMC says ‘no’ to Uptravi

Selexipag is an innovative, oral treatment that specifically targets the prostacyclin pathway.

Actelion Pharmaceuticals UK has announced that the Scottish Medicines Consortium (SMC) has issued guidance not recommending the use of selexipag (Uptravi) in Scotland for a sub-population of adult patients with pulmonary arterial hypertension (PAH) – a potentially life-threatening condition that causes an increase in blood pressure between the right side of the heart and the lungs. Specifically, the decision has been made in relation to patients with functional class III PAH (moderate-severe) who are insufficiently controlled with dual oral combination therapy and who would otherwise be considered for treatment with inhaled iloprost.

"It is disappointing to us and our patients that we have not been given approval to prescribe selexipag," commented Dr Martin Johnson, Consultant Physician at the Scottish Pulmonary Vascular Unit, Clydebank. "This medication has efficacy proven in a large clinical trial, has been licensed in the US and Europe and is already being used by my colleagues in other countries. There is no other oral prostacyclin pathway agent available to us in the UK and this mode of drug delivery, oral, has distinct advantages for patients over the current alternatives, such as nebulised."

Selexipag is an innovative, oral treatment that specifically targets the prostacyclin pathway – an important pathway involved in the development of PAH. It has been shown to improve long-term outcomes for PAH patients in a Phase III clinical study of over 1,000 patients, the largest randomised controlled trial ever conducted in PAH. Data from the GRIPHON trial, which supported the SMC submission, showed a significant 40% overall reduction in the risk of a composite morbidity-mortality event in PAH patients treated with selexipag compared to placebo. This was driven by a reduction in hospitalisations for PAH worsening and other events resulting from disease progression. The effect of selexipag treatment was consistent in the sub-group of patients already receiving dual oral combination therapy for PAH (approximately a third of patients in the study). In the study, selexipag was generally well-tolerated by patients, with the majority of commonly reported side effects being mild to moderate.

"We are very disappointed with the SMC’s decision, which will be a huge blow for those living with PAH" commented Dr Iain Armstrong, Chair of the patient organisation, Pulmonary Hypertension Association (PHA UK). "PAH impacts every aspect of a person’s life – their physical, social, emotional and psychological wellbeing. It is vital that those affected by PAH have access to new treatments that could improve their ability to engage in the normal day-to-day activities that most of us take for granted. There may be a significant number of patients with PAH who can’t use the currently available prostanoid treatments and for these patients, selexipag – an easier to manage, oral treatment – could help to keep them well and out of hospital for longer. I would urge the SMC and Actelion to continue working together to find a solution and ensure that access for patients in Scotland is secured as quickly as possible."

The negative decision by the SMC comes despite recognition that data submitted provided strong clinical evidence for selexipag. A problem cited was the fact that the committee was unable to assess the long-term benefit of selexipag in comparison with inhaled iloprost (the comparator defined in the submission). However, this was due to a lack of long-term available data for inhaled iloprost – long-term data for selexipag was included in the SMC submission.

"We are extremely disappointed that the SMC has denied access to selexipag for this group of PAH patients in Scotland, who remain in need of new options to help them better manage their disease," commented Robin Bhattacherjee, General Manager, Actelion Pharmaceuticals UK. "This decision comes despite recognition of the significant benefits that this treatment brings to patients and our provision of a confidential discounted price through a patient access scheme aimed at getting this medicine to those in need as quickly as possible. Actelion is fully committed to continuing to work with the SMC to find a solution, and we urge further discussion on this issue so that a solution can be found."