Now for the triple...
Over the last few weeks deals have been announced in each of the nations of the UK that ensure that the cystic fibrosis drugs Orkambi and Symkevi join Kalydeco in being available to all eligible people on the NHS.
This is approximately 5,000 children and adults, which represents about half of the people with CF across the UK.
The wait for these drugs has been far, far too long and real harm has been done, and I fear we would still be waiting if it wasn’t for the passion and commitment of the whole CF community. No one with cystic fibrosis must be made to endure this wait again.
In the midst of news of the deals came the announcement that the Food and Drug Administration (FDA) in the United States had approved Vertex’s triple combination therapy (known in the US as Trikafta).
The hopes we had for this treatment, which I wrote about over the summer, have been realised with the latest published results. The data shows stunning impact on lung function, and critically that the drugs can help a far wider group of people with CF than other treatments. We estimate that this could be approximately 9,000 people, or 90% of those with the condition.
Orkambi and Symkevi are important, but the triple combination therapy has been described as truly “game-changing”.
That is why as the drug begins its regulatory journey through the European Medicines Agency (EMA) we must do all we can to ensure rapid and comprehensive access. We must not fall behind again.
We are talking to the key parties to ensure that everything possible is done and that there are no excuses for delay. In this time of political uncertainty it is vital that we keep politicians engaged by getting them to commit to the pledge that the UK will lead the world in access to new cystic fibrosis medicines.
I also want to say that we are painfully aware that not everyone will stand to benefit even from the triple combination therapy. For 10% of people living with CF in the UK there is currently no appropriate therapy. That is why we are investing in cutting-edge research as part of the global mission to create treatments that will work regardless of genotype.
No one must be left behind.
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