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Cystic Fibrosis Trust

Orkambi

Orkambi (ivacaftor/lumacaftor) is the second precision medicine to be licensed for use in the UK by people with cystic fibrosis, although it is not currently provided by the NHS except in rare cases on compassionate grounds.

We are planning some new Orkambi campaign actions for the end of 2017. Scroll down to find out more.

What you need to know about Orkambi

The first precision medicine for cystic fibrosis was Kalydeco. Orkambi is the second such drug to be licensed for use in people with cystic fibrosis.

  • Orkambi treats the F508del mutation, which around 50% of people with cystic fibrosis in the UK have.
  • Orkambi is a combination medicine, made up of Kalydeco (ivacaftor) and Lumacaftor. The Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs.

Who is currently receiving Orkambi in the UK?

  • According to the 2014 UK Cystic Fibrosis Registry Report, there are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland who could benefit from Orkambi.
  • Orkambi has been licensed for use in the UK for people with CF over the age of 12 who have two copies of the F508del mutation. Despite this, the National Institute for Health and Care Excellence (NICE) has rejected its use on the NHS.
  • In May 2016, the Scottish Medicines Consortium (SMC) noted that Orkambi was a beneficial treatment. In June 2016, NICE recognised Orkambi as an important treatment.
  • Both the SMC and NICE were unable to recommend Orkambi for general use within the NHS/HSCNI on grounds of cost-effectiveness and a lack of long-term data.
  • Orkambi is currently only prescribed to people on compassionate grounds. Compassionate use means that Vertex Pharmceuticals - the manufacturer of Orkambi - provide the drug to people who fulfil a number of criteria. Contact your clinician for more information on compassionate use.

What effect does Orkambi have?

96-week data shows that Orkambi can slow decline in lung function – the main cause of death among people with cystic fibrosis - by 42%. This data was unavailable to NICE when they appraised Orkambi.

Why is it not available to everyone who needs it?

The processes involved in making drugs available to the people they could help are complicated. Creating and testing a drug like Orkambi is very expensive and a lack of long-term data can make it hard for NICE and the SMC, the bodies that appraises these drugs for use in the NHS/HSCNI, to accurately assess their cost-effectiveness in order to make them available.

What are we doing about it?

Stay up to date with Stopping the Clock, our dedicated campaign to put pressure on the Government, NHS and pharmaceutical companies to ensure these drugs reach the people who need them without delay.


Next steps

We've been working hard to capitalise on the momentum of the protests in June, and there are a number of actions we would like you to get involved in in your area in the upcoming months.

Northern Ireland

We’ve drafted a cross-party letter to the Direct Rule Minister for Health, Jeremy Hunt, and Department of health Permanent Secretary, Richard Pengelly, calling for a timely resolution to the ongoing negotiations between Vertex and Health and Social Care Northern Ireland.

These letters have been signed by 66 MLAs from a number of different parties and sent to Jeremy Hunt and Richard Pengelly. Read the letters here:

Wales

We have released a petition to the Welsh Assembly which calls for a resolution to negotiations between NHS Wales and Vertex. If the petition gets over 5000 signatures, it will automatically be considered for a debate at the Welsh Assembly. Anyone can sign the petition, no matter where they are in the UK, so get signing now!

Sign the petition

England

We have organised a parliamentary event that will held on Wednesday 29 November from 8.30–10.30am at the Churchill Room, Palace of Westminster,and is being sponsored by Ian Austin, MP for Dudley North. This event will be a great opportunity to get MPs from all political parties in one room with representatives from the Trust, Vertex and campaigners.

We’re asking you to get in touch with your MP and ask that they attend the event. Please download our template letter and use it to get in touch with your MP. Don’t forget to copy our Public Affairs Team into the email so that we can follow up with your MP.

Download the template

If you’d be interested in attending the event yourself please contact us at publicaffairteam@cysticfibrosis.org.uk. There’s a limited number of places, which will be allocated on a first come, first served basis.

Scotland

We hope to secure a debate at the Scottish Parliament to discuss Orkambi and the wider issue of access to medicines in Scotland, in relation to the Montgomery review. Check back here in the next few weeks for further details.

For the latest news on our Stopping the Clock campaign and other updates in the CF community, sign up to receive our e-newsletter.

It's personal!

Precision medicines like Orkambi are an example of how healthcare is becoming increasingly tailored to the individual, rather than the whole population of people with cystic fibrosis in the UK.

What's personalised healthcare?

Other precision meds?

Kalydeco is the first precision medicine for people with cystic fibrosis and is available for some people in the UK on the NHS. Find out how it works, who can have it and why it's different to Orkambi.

What is Kalydeco?

Why it matters?

Cystic fibrosis places limits on every aspect of your life. Use our interactive body to explore the effects it can have from head to toe, and see why it's vital to get access to precision medicines without delay.

How CF affects the body