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Cystic fibrosis drug given green light in England

Child with cystic fibrosis in a hospital bed Image copyright Getty Images

A life-extending drug for cystic fibrosis will be available on the NHS in England, health bosses say.

NHS England reached a deal with Orkambi manufacturers Vertex Pharmaceuticals after months of talks. Patients should be able to get the drug within 30 days.

The drug improves lung function and reduces breathing difficulties and can be given to children as young as two.

The firm wanted to charge £100,000 per patient per year but a compromise has been reached in a confidential deal.

It is understood to involve significantly less than the sum originally asked for.

‘Best present’

Patients and campaigners have expressed delight at the announcement.

Christina Walker said it meant her son, Luis, eight, should be on it by Christmas, calling it the “best present ever”.

“I can’t stop smiling. I’m overwhelmingly happy. It’s absolutely wonderful,” she said.

The deal comes after the Scottish Government reached an agreement with the manufacturers last month.

Wales and Northern Ireland can now also access the drug under the same terms negotiated by NHS England.

‘Patients and taxpayers’

Two other drugs made by Vertex – Symkevi and Kalydeco – will be made available as part of the deal.

These also treat cystic fibrosis symptoms but cannot be used until a patient is 12.

The treatments do not work for all patients with cystic fibrosis – only those with certain mutations.

Image caption More than 10,000 people in the UK have cystic fibrosis, which cause fatal lung damage

It is estimated about half of the 10,000 patients in the UK will benefit from these drugs.

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage.

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

NHS England chief executive Simon Stevens said the deal was “good for patients and fair to British taxpayers”.

Health Secretary Matt Hancock described it as “wonderful news”.

Source: bbc.co.uk

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