As Vertex's statement notes, the application was supported by positive results of two global trials in people with cystic fibrosis (CF) with either two copies of F508del or a single copy of F508del and one minimal function mutation. Both of these trials showed significant improvements in lung function, a decrease in exacerbations and an increase in reported quality of life.
This news comes after Vertex gained approval for Trikafta in the United States from the Food and Drug Administration (FDA), making it potentially available to people with CF across the country.
It should be noted that the EMA process can take many months. The Cystic Fibrosis Trust is working hard to understand the most likely timetable, how long it may take for the drugs to be made available and what can be done to speed up these processes, and will keep the CF community updated on any developments.
David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said: “This is another very significant day for people with CF across the UK. We know that the months ahead are likely to bring both new and familiar challenges and we will not stop until everyone has access to the best drugs that exist."
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