What is Symkevi?
Symkevi is a combination of ivacaftor and tezacaftor. The treatment has been shown to be effective in people with CF with two copies of the F508del mutation (which around 50% of people with CF in the UK have) and those with one copy of F508del and another residual function mutation (approximately 5% of people with CF in the UK): P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.
What effect does Symkevi have?
In the first study into Symkevi, 510 patients with cystic fibrosis with two copies of the F508del gene took Symkevi with ivacaftor, or took a placebo. After 24 weeks of treatment, patients who took Symkevi and ivacaftor had an average increase in FEV1 (forced expiratory volume – a measure of lung function) of 3.4% compared with a reduction of 0.6% in patients who took the placebo.
In the second study 248 patients with cystic fibrosis with one copy of the F508del mutation and one of the residual function mutations (listed above) took Symkevi with ivacaftor, ivacaftor on its own, or a placebo. Lung function was measured after 4 weeks and 8 weeks of treatment. Patients who took Symkevi and ivacaftor had an average increase in FEV1 of 6.5% compared with an increase of 4.4% in patients who took ivacaftor alone and a reduction of 0.3% in patients who took placebo.
Is Symkevi available in the UK?
Symkevi is available for use in the UK for people with CF aged 12 and over with two copies of F508del or one F508del and a residual function mutation (see above).
Managed access agreements to make Symkevi available on the NHS have now been agreed in England, Scotland, Wales and Northern Ireland. To find out whether you or your child might be eligible for a precision medicine, please speak to your CF team.
The terms of the NHS England deal for Kaftrio also mean that as many as 300 patients with some rarer genetic mutations, which fall outside of the scope of the European licensing body’s considerations, will now be able to benefit from Kalydeco and Symkevi. The list of rare mutations now eligible for these modulators can be found here.
Equivalent terms for rare mutation access for people with CF in Wales, Northern Ireland and Scotland were ensured by NHS England. We are currently seeking confirmation that expanded access to Kalydeco and Symkevi is available throughout the UK.
The UK CF Registry, which is managed and sponsored by the Trust, is playing a vital role in supporting these agreements by collecting data on the health outcomes of individuals taking Symkevi and other recently licensed drugs.
Find out about our fight for access to life-saving drugs, as well as how you can help to support our work.