Access to Orkambi and Symkevi across the UK
After a tireless campaign by the Cystic Fibrosis Trust and the CF community, England, Wales, Scotland and Northern Ireland now have access to all currently-licensed CF precision medicines: Kalydeco, Orkambi and Symkevi.
We are now focussing our efforts on the latest Vertex precision medicine, the triple combination therapy (known as Trikafta in the United States). Find out where the drug is at in the licensing process, who it could stand to benefit and what we are doing to support the process.
Timeline for the triple combination therapy
On Thursday 20 February, the National Institute for Health and Care Excellence (NICE) announced its planned timeline for assessing the triple combination therapy and we also learnt that the EMA are to allow more time in their process to assess who the license would cover. Find out what this means and watch a Facebook Live video from Nick Medhurst, the Head of Policy and Public Affairs, here.
Find out more
CF Europe letter to the EMA
This month CF Europe wrote to the EMA regarding their assessment of the triple combination therapy. The letter set out that, for many people with cystic fibrosis in Europe, being on the precipice of having access to this therapy represents a major milestone in our broader mission to beat cystic fibrosis.
The letter emphasises the results of the two major clinical trials for the triple therapy show improvement of an unprecedented magnitude that could benefit the majority of people with cystic fibrosis in Europe. The letter implores the EMA to take a pragmatic approach to awarding a broad licence to the triple combination therapy, while maintaining a sense of urgency in their assessment that reflects the heightened anticipation many feel about this life-saving treatment.
CF Europe offered to support the EMA in this process by offering the expertise of the CF community, from patients and families, to clinicians and researchers across Europe, whose insight will be valuable as the EMA make their assessment.
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