Cystic fibrosis drug given green light in England

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By BBC Health News

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

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

The drug improves lung function 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 be significantly less than the sum originally asked for.

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

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

‘Good for 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.

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 around half of those with the condition live to celebrate their 40th birthday.

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” that would improve.