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Hundreds set to benefit from new cystic fibrosis drugs

Orkambi and Symkevi will be free to use for those dealing with the illness across Scotland.

Cystic Fibrosis: Doctors will be able to prescribe new drugs.
Cystic Fibrosis: Doctors will be able to prescribe new drugs.

Two new drugs to treat cystic fibrosis will be made available on the NHS in Scotland, potentially benefiting around 400 patients.

Orkambi and Symkevi will be free to use for those dealing with the illness across the country after the Scottish Government signed a five-year deal with US pharmaceutical firm Vertex.

Cystic fibrosis campaigners hailed the decision to approve the drugs for use by the Scottish health service as a “landmark moment” and urged the rest of the UK to do the same.

Orkambi is used to treat cystic fibrosis in patients aged two and older while Symkevi treats those aged 12 and up, with both designed to improve lung health.

Last month, both drugs were rejected for routine use by the Scottish Medicines Consortium (SMC) over doubts on their cost-effectiveness.

But the decision was reversed after Scottish ministers negotiated a five-year pricing agreement with Vertex to supply the medication.

Cystic fibrosis is an inherited, life-shortening illness that creates a mucus build-up which causes chronic lung infections and progressive lung damage.

Neither drugs are a cure, but Symkevi has been shown in studies to improve lung capacity in cystic fibrosis patients, while Orkambi slows decline in lung function – the main cause of death for cystic fibrosis patients.

A further effect of both of the drugs, proponents say, is to reduce the amount of hospital admissions patients need.

Around 900 people in Scotland live with cystic fibrosis, with the Scottish Government estimating that about 400 may be suitable for treatment with the new medicines.

Health secretary Jeane Freeman said: “This is fantastic news for those living in Scotland with cystic fibrosis and their families.

“This new pricing agreement with medicines manufacturer Vertex Pharmaceuticals, secured by the Scottish Government, will allow more people with cystic fibrosis to benefit from medicines Orkambi and Symkevi and live fuller lives for longer.

“The agreement has been reached after extensive discussions between the Scottish Government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount.”

Vertex senior vice president Ludovic Fenaux 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.”

The Cystic Fibrosis Trust welcomed the decision but called on health authorities across the UK to do likewise.

Chief executive David Ramsden said: “This is a landmark moment for the hundreds of people with cystic fibrosis and their families across Scotland who have tirelessly campaigned for years to access these drugs.

“This breakthrough is a victory for their perseverance and enduring hope.”

He added: “We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland.

“Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery.

“Scotland’s success must now be replicated across the UK without further damaging delay.”


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