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Benefits of Kalydeco (ivacaftor) for preschoolers

New research highlights importance of access for young children with specific CF mutations.

Children aged two to five with cystic fibrosis could be missing out on improved pancreatic function, weight gain and movement of salt between cells because they are not receiving Kalydeco (ivacaftor), according to new research published in ‘The Lancet Respiratory Medicine’ journal.

The 33 pre-school children who completed the six-month trial experienced significant reductions in sweat chloride levels, suggesting a partial correction of the underlying cause of cystic fibrosis, a lack of control over the movement of salt in and out of cells.

The study also showed improved weight gain and, in a quarter of the children, a dramatic improvement in pancreatic function, above the level clinically deemed insufficient.

The report’s authors described this discovery, the first shown with any cystic fibrosis drug in subjects, as “a window in early life where at least partial restoration of pancreatic function may be possible”.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, said: “This research underscores how vital it is that people with cystic fibrosis are given access to transformational treatments. We are determined to ensure that people with cystic fibrosis have access to life-changing treatments as soon as possible.”

Ivacaftor is currently available for those aged six and over with one of nine gating mutations, around five per cent of the UK cystic fibrosis population.

Ivacaftor was recently licensed by the EU for children aged two to five years old with one of nine gating mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R).

As part of the Stopping the Clock campaign, the Trust is campaigning to ensure that as many people with CF as possible have access to precision medicines by 2020. The Policy Team is pressing the drug’s manufacturer and the NHS to make this treatment available to young children.

Could your child benefit from today’s announcement? Find out at GenotypeMatters.org