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.
On 13 November, the Welsh Minister for Health and Social Services, Vaughan Gething, announced that the Welsh Government has agreed terms in principle with Vertex Pharmaceuticals to make Orkambi and Symkevi available alongside Kalydeco in Wales. We await further confirmation on the details of this deal but understand that the Government aims to have it finalised by the end of November and intends to begin to roll out access where clinically appropriate to eligible patients the following month. To find out whether you or your child might be eligible for a precision medicine, please speak to your CF team.