Two “life-changing” cystic fibrosis drugs are to be made available to about 400 patients in Scotland – a month after their use was rejected...

Two “life-changing” cystic fibrosis drugs are to be made available to about 400 patients in Scotland – a month after their use was rejected by the NHS.

Pharmaceutical company Vertex has announced it has signed a five-year deal with the Scottish government.

Orkambi and Symkevi were rejected for routine use by NHS Scotland in August.

The drugs help improve lung health, but were to cost about £100,000 per patient, per year.

Ludovic Fenaux, Vertex senior vice president said: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.

“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi and Symkevi.”

As part of the five-year agreement, US-based Vertex has also committed to collecting data on the medicines to support any future submissions to the Scottish Medicines Consortium (SMC).

The SMC previously rejected the use of the drug saying there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs.

Some Scottish patients have previously had access to Orkambi and Symkevi through the Peer Approved Clinical System Tier 2 (PACS Tier 2), which allows doctors to apply for access on behalf of individual patients.

What are Orkambi and Symkevi?

Since 2015, the drug Orkambi has been licensed to treat cystic fibrosis in patients from two-year-olds to adults, who have a specific genetic mutation known as F508del.

It was not available on the NHS, except for certain people on compassionate grounds.

Symkevi is used to treat the same mutation in patients age 12 and older.

The mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Orkambi has been shown in clinical trials to improve lung function and respiratory symptoms in people with cystic fibrosis.

It is the first of a string of drugs that have been developed, with newer ones expected to be even more effective.

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage, and affects about 10,400 people in the UK – around 900 of them in Scotland.

Only around half of those with the condition live to the age of 40.

NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with cystic fibrosis.

Earlier this year campaigners appealed to the UK government to use its powers to break a deadlock in making Orkambi available on the NHS in England.

Vertex refused a £500m offer for the drug over five years – which was described as the “largest commitment” NHS England had ever made.

Those affected want other drug firms to be asked to make a cheaper version.

The UK government’s Department of Health said its approach was still to urge Vertex to accept NHS England’s “generous offer”.

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