Patients with a rare form of cystic fibrosis in England and Wales are currently being denied access to Vertex’ “life-changing” treatment Kalydeco (ivacaftor) on the National Health Service, even though it has regulatory backing and is already used for the most common mutation of the disease.

Kalydeco is currently ‘routinely’ available to around 360 patients with the G551D mutation of CF and, according to the Cystic Fibrosis Trust, new data shows the drug is “resulting in a dramatic increase in lung function and a reduction in hospital admissions”.

In July, European regulators expanded its label to include the much rarer non-G551D gating mutations, but near seven months later patients are still waiting to whether they will be able to get NHS access to the therapy, causing anger and frustration.  

“I’ve seen first-hand the rapid and life-changing results that Kalydeco has on CF sufferers and the thought of not being able to receive it now that I know I am suitable is beyond comprehension,” commented 30 year-old patient Mike Boyle.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, called it “indefensible” that CF patients are being denied access to “the only treatment which provides them with both a long-term quality of life and survival benefit”, and noted that the drug could normalise life expectancy, “meaning that for the first time patients face the prospect of dying from old age, not CF, where the median age of death currently stands at 27”.

Jason McCartney MP, Chair of the All-Party Group on CF, is urging NHS England and Vertex “to find a way to extend the gift of life to these 44 people, for who time and hope is running out”.