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Cystic Fibrosis Trust

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).


What is Symkevi?

Like Orkambi, which combines ivacaftor with lumacaftor, this new, 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 mutation that has residual function. The results have been positive.

How have studies progressed?

Two global Phase III trials have revealed that Symkevi could reduce the effects of cystic fibrosis on people with two copies of the F508del mutation and present significant improvements for those with one F508del and one ‘residual function’ mutation.

EVOLVE study

The EVOLVE study, which involves those with two copies of the F508del mutation found that the treatment was more stable than Orkambi, with similar benefits of around 4% lung function improvement and fewer side effects.

EXPAND study

The EXPAND study, which involves those with one F508del and one residual function mutation found an average absolute improvement in FEV1 lung function of 6.8% compared with the placebo.

Next steps

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

Symkevi is not routinely available in any of the four UK nations. Talks have been ongoing between the drug’s manufacturer, Vertex Pharmaceuticals, and the health bodies and decision makers in each nation.

In August 2019, the Scottish Medicine Consortium (SMC) announced it was not able to recommend Symkevi (and similar drug, Orkambi) for routine use by NHS Scotland, adding that the company had failed to justify the price they were offering. Applications to use Symkevi in Scotland can still be made on an individual, case-by-case basis, under the PACS Tier 2 system and on compassionate grounds.

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.

Stopping the Clock

Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines.

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