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.

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.