Actelion says it is disappointed with a decision by Scottish cost regulators to refuse NHS funding for pulmonary arterial hypertension drug Uptravi.
The Scottish Medicines Consortium (SMC) has issued guidance not recommending use of Uptravi (selexipag) for a sub-population of adult patients with the potentially life-threatening condition, which causes an increase in blood pressure between the right side of the heart and the lungs.
The decision relates 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.
PAH is a severe, progressive and life-shortening disease affecting hundreds of people across Scotland.
Uptravi, which specifically targets the prostacyclin pathway involved in the development of PAH, has been shown to improve long-term outcomes for PAH patients; in the Phase III GRIPHON trial there was a 40 percent overall reduction in the risk of a composite morbidity-mortality event in PAH patients treated with the drug compared to placebo.
“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.”
According to the firm, “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 very disappointed with the SMC’s decision, which will be a huge blow for those living with PAH,” added Dr Iain Armstrong, chair of patient organisation, Pulmonary Hypertension Association (PHA UK).
“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.”
Actelion stressed that it remains “fully committed” to continuing work with the SMC to find a solution, urging “further discussion on this issue so that a solution can be found”.
In more positive news for patients, the SMC did issue a green light for: AstraZeneca’s Qtern, a fixed-dose combination of saxagliptin and dapagliflozin in a single pill, for restricted use for type II diabetes; MSD’s Keytruda (pembrolizumab) for advanced non-small cell lung cancer (NSCLC); Bristol-Myers Squibb’s Opdivo (nivolumab) for Hodgkin lymphoma; and Janssen-Cilag’s Stelara (ustekinumab) for the treatment of Crohn’s Disease.