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New cystic fibrosis drug approved for UK use

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Orkambi approved for UK use Cystic fibrosis affects almost 10,000 people in the UK

Orkambi, a drug that improves lung function in cystic fibrosis patients, could improve the lives of almost 4000 people in England.

NHS England have now reached a deal with the manufacturers of Orkambi, Vertex Pharmaceuticals, that makes this drug available to patients in England. The deal also allows Wales and Northern Ireland to access this drug for the same price, though they have not yet announced if they intend to do so. NHS Scotland made a similar deal with Vertex Pharmaceuticals in September.

'The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis,' said Simon Stevens, chief executive of NHS England.

'That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS.'

Orkambi treats patients with a specific mutation (F508del) and it’s estimated by the Cystic Fibrosis Trust that around 50% of cystic fibrosis patients in the UK possess this mutation. Orkambi can be given to patients as young as two years old and is therefore set to improve the lives of many patients in the UK.

Matt Hancock, Health Secretary, commented on the deal, saying ‘The deal is great value for money for the NHS, and crucially, will improve thousands of lives. This deal- on the back of several others this summer- shows why we get some of the best value drugs in the world, and is another reason to be so proud of our NHS’.

The other deals recently reached include drugs for multiple sclerosis, hepatitis C and lung cancer.

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