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Cystic Fibrosis Trust

Other precision medicines

Read about two new precision medicines, Symkevi and Vertex Pharmaceuticals' triple combination therapy (Trikafta in the United States), and find out how they could help to treat people with cystic fibrosis (CF).


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. Six weeks later NHS England announced that people with CF in England will receive access to the drug as part of a two-year managed access agreement with Vertex, and shortly afterwards the Department of Health in Northern Ireland has confirmed its intention to complete a similar deal, the plans for which are now being finalised

However, the drug is still not routinely available for use within Wales on the 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 accessCompassionate 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, 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.

What is Vertex's triple combination therapy?

The triple combination therapy (brand name Trikafta in the United States) uses a combination of ivacaftor, tezacaftor and elexacaftor. The drug was tested in clinical trials on two groups: people with one copy of the F508del mutation and one copy of a 'minimal function mutation', and people with two copies of the F508del mutation, and both trials produced positive results. In the United States, a licence has been granted by the FDA for anyone over the age of 12 with one copy of F508del, regardless of their second mutation. This accounts for approximately 90% of the CF population.

When will it become available?

Recently, the triple combination therapy was approved in the United States for those aged 12 and over who have at least one F508del mutation. Vertex have submitted the drug to the EMA, who are analysing it for safety and clinical effectiveness, with further updates expected in 2020.

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