Cystic fibrosis drug Orkambi to be available free on the NHS

On October 24, 2019, NHS England reached a deal with the manufacturers Vertex Pharmaceuticals to make all three of their UK-licensed cystic fibrosis medicines available free on the NHS.

Orkambi the life-extending drug for cystic fibrosis (CF) patients is finally available through NHS England.

‘Wonder drug’ Orkambi

Cystic fibrosis is a genetic condition that can cause fatal lung damage.  Orkambi is perceived as a ‘wonder drug’ because it helps improve lung function and reduces breathing difficulties and can be prescribed to patients age two years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene.

Cystic fibrosis drug ORKAMBI manufactured by Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals wanted to charge £100,000 per patient per year but a compromise has been reached in a confidential deal. Two other drugs made by Vertex – Symkevi and Kalydeco – will be made available as part of the deal.

These also treat cystic fibrosis symptoms. Symkevi is restricted to over 12-year-olds, while Kalydeco can be used from 12 months.

Around 5,000 people in England will now have full access to the drugs Orkambi, Symkevi and Kalydeco and there is no cap on patient numbers.

‘Good for patients and taxpayers’

Patients and campaigners are understandably delighted with the resolution.

Announcing the agreement, the BBC reportedNHS Chief Executive Simon Stevens said: “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.

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

“I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”

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

David Ramsden, of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.”

He also said more good news could be coming soon as a new therapy Trikafta, which 90% of people with the condition could benefit from, was getting close to being licensed for use.

Orkambi could benefit around 5000 people

Orkambi (a lumacaftor/ivacaftor combo) has been formulated to counter the F508del mutation, the most common of CF-causing mutations in the CFTR gene. It should not be used in patients other than those who have two copies of the F508del mutation in their CFTR gene. It is estimated that it can be prescribed to around 50% of CF patients in the UK – around 5,000 people in total. Lumacaftor is a corrector, and brings the defective chloride channel protein to the cell surface where they can be, while ivacaftor is a potentiator, and facilitates the opening of these channels so they can transport ions more effectively.

This website is for informational purposes only and is not intended to be a substitute for professional medical advice. You should talk to your healthcare provider about whether ORKAMBI is right for you.