PATIENTS in Scotland with an incurable lung condition will be the first in the UK to get a new life-extending drug on the NHS.
The Scottish Medicines Consortium has given the go ahead for selexipag to be prescribed on the NHS to patients with pulmonary arterial hypertension (PAH).
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The condition causes an increase in blood pressure in the blood vessels between the right side of the heart and the lungs.
Selexipag – also known by the brand name Uptravi – will be made available on a long-term basis to individuals who are at stage three of the disease, when even everyday tasks like housework, cleaning and climbing the stairs have become exhausting, and who are not responding to existing treatments.
Left untreated, patients at stage three live only two and a half years on average.
The new drug, which comes as a tablet which can be taken at home twice a day, is expected to benefit around 11 patients during the first year and 42 by year five.
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It has been shown in clinical trials to slow progression of the disease and significantly improve quality of life, in some cases allowing patients to return to work.
PAH disproportionately affects women and it tends to strike patients between the ages of 20 and 60.
Early symptoms include breathlessness, fatigue, dizziness and chest pain during exercise which is often mistaken for being unfit or asthma.
However, as the illness progresses, these symptoms are experienced during rest and can result in patients becoming largely housebound.
It is particularly devastating as patients with PAH are advised against becoming pregnant due to the high risk of complications, yet the majority of sufferers will experience onset in their 30s and 40s at an age when many will have young children or want to start a family.
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Dr Martin Johnson, consultant physician at the Scottish Pulmonary Vascular Unit, said he was “delighted” by the decision.
He added: “This will increase the options open to people with PAH in Scotland and represents a significant step forward in how we can treat this disease.”
In Scotland, there are currently 620 patients being managed for PAH at the Golden Jubilee Hospital in Clydebank, near Glasgow, which is Scotland’s only specialist centre for the condition.
Selexipag is the first oral medication which targets one of the three key pathways – the prostacyclin pathway – known to play a role in the development of PAH.
Prostacyclin is a signalling molecule in the human body which instructs blood vessels to relax and prevents the build up of blood platelets in the arteries.
Current therapies which target this pathway, although effective, can be difficult for patients to administer since they involve intravenous infusions in hospital or inhaling drugs via nebulisers up to nine times a day – including overnight.
The latest decision by the SMC reverses its previous rejection of the drug in July 2017.
Dr Iain Armstrong, Chair, Pulmonary Hypertension Association UK, said: “PAH is not just a physical condition; it has a huge impact both psychologically and socially, often when patients are in the prime of their life and family roles.
“Whilst this is excellent News for Scottish patients, we are mindful that just across the border in England and Wales, NHS patients are still waiting for this much-needed medicine.”
Robin Bhattacherjee, General Manager of the drug’s manufacturer, Actelion Pharmaceuticals UK & Ireland, added: “For people living with this devastating and progressive disease, every day matters and a medication like selexipag has been much needed for a long time.
“We are pleased that the SMC and Actelion were able to work to ensure these patients have access.
“Our hope is that all eligible patients across the UK and Ireland will soon be able to routinely benefit from selexipag on the NHS and we will continue to work with all relevant parties to make this a reality.”
Source : HeraldScotland