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Other precision medicines

In February 2018, drug company, Vertex Pharmaceuticals, have recently released promising results from studies into two new types of ‘combination therapy’. These are treatments that use two or three drug compounds in combination to treat cystic fibrosis. Find out more about the treatments and how they could affect people with cystic fibrosis (CF).

Symkevi

What is Symkevi?

Like Orkambi, which combines ivacaftor with lumacaftor, this dual combination therapy ​(brand name Symkevi) uses two drugs together in one treatment. Symkevi combines ivacaftor with new drug compound tezacaftor. The treatment has been shown to be effective in people with CF with two copies of the F508del mutation and those with one copy of F508del and another residual function mutation: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.

Is Symkevi available in the UK?

The European Medicines Agency (EMA) has granted marketing authorisation for Symkevi for those aged 12 and over with two copies of F508del or one F508del and a residual function mutation.

In September 2019 the Scottish Government announced a five-year deal giving access to the drug for everyone with CF in Scotland who can benefit from it.

However, the drug is still not routinely available for use within the NHS across the rest of the UK on grounds of cost-effectiveness and a lack of long-term data, and the drug is currently only prescribed to people on compassionate grounds and through limited clinical trial access.

Compassionate use means that Vertex Pharmaceuticals provide the drug to people who fulfil a number of criteria. 

To find out whether you or your child might be eligible for a precision medicine, whether in Scotland or through compassionate use, please speak to your CF team.

Triple combination therapies

What are triple combination therapies?

Triple combination therapies are treatments that work to help correct a series of complications at the cellular, root-cause of cystic fibrosis. At least three companies, including Galapagos Therapeutics, Proteostasis Therapeutics and Vertex Pharmaceuticals, are developing potential triple combination therapies.

Vertex Pharmaceuticals has recently completed Phase II clinical trials of a series of triple combination therapies, which include drug compounds ivacaftor, tezacaftor and one of three new, additional compounds, and could work in people with one copy of the F508del mutation and one copy of a 'minimal function mutation' - approximately 90% of the CF population.

How have studies progressed?

In February 2018, Vertex Pharmaceuticals announced its plans to progress two types of triple combination therapy to Phase III trials. These trials will test these triple combinations on people with CF with one copy of the F508del mutation and a ‘minimal function’ mutation, and those with two copies of the F508del mutation.

For more information about the different trials, read our summary here.

Next steps

Details of these Phase III trials, including when and where the studies will start, have yet to be announced. To participate in clinical trials head to the Trials Tracker, or find out more about how you can get involved in vital research on our Clinical Trials Digital Hub.

When will these treatments become available?

While results look promising, neither of these treatments are currently available on the NHS. As part of our campaign for access to precision medicines we will continue to put pressure on the Government, NHS and pharmaceutical companies to ensure that these new generation therapies, as well as already licensed drugs like Orkambi and Kalydeco, can reach the people who need them without delay.






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