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New treatment Symkevi gets greenlight for Europe

The European Medicines Agency (EMA) has today given marketing authorisation for new cystic fibrosis (CF) medicine Symkevi. This important milestone means that, if an agreement can be reached between the NHS and the manufacturer, Vertex Pharmaceuticals, the treatment could be available for people with CF in the UK.

Symkevi is a combination of two drugs (ivacaftor and tezacaftor) that when taken once a day, alongside another dose of Kalydeco (ivacaftor), has been proven to improve lung function and reduce infection exacerbations, which often lead to antibiotic therapy and hospitalisation.

Like Orkambi, Symkevi (in combination with Kalydeco) has proven effective for people who have two copies of the F508del mutation, although Symkevi is only licensed for people aged 12 and older, whereas Orkambi has proven effective for children as young as two.

Additionally, Symkevi can be used to treat more mutations than Orkambi or Kalydeco alone, with 14 so-called ‘residual-function’ mutations responding to treatment if the person with CF also has a copy of F508del:

P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T

Symkevi is the third precision medicine to gain a European license, after Kalydeco (ivacaftor) and Orkambi (lumacaftor-ivacaftor) and further expands the number of people in the UK who could benefit from a medicine that treats the root cause of CF, rather than manage symptoms.

Trust Chief Executive David Ramsden has demanded immediate action to ensure access to Symkevi for all who could benefit, as soon as possible: “With Orkambi still denied to people with CF in the UK, despite reaching this milestone nearly three years ago, we are calling on Vertex to fully engage immediately with both the Scottish Medicines Consortium (SMC) and the National Institute of Health and Care Excellence (NICE), who together provide advice on the use of cystic fibrosis medicines for everyone in the UK.

“We acknowledge ongoing discussions between Vertex and representatives of the Scottish Government, NHS England and NICE regarding access to medicines. However, we call on all parties to bring these discussions forward to a positive resolution.”

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