‘Groundbreaking’: Cystic fibrosis drug is safe and effective in newborns, new research shows
A “groundbreaking” drug which targets the underlying cause of cystic fibrosis has been shown to be safe and effective in newborns aged four months or older.
Cystic fibrosis (CF) is a life-limiting genetic condition that affects around 10,500 people in the UK. It causes thick, sticky mucus to build up in the lungs and other organs, leading to a range of health problems.
Kalydeco (ivacaftor) is a drug that was previously only available to babies aged four months or older. However, a new study has shown that it is also safe and effective in newborns aged four weeks or older.
The study, which was published in the journal The Lancet Respiratory Medicine, involved 77 babies with CF. The babies were given Kalydeco for either 24 weeks or 48 weeks.
The results showed that Kalydeco was safe and well-tolerated in all of the babies.
- There were no serious side effects.
- The babies showed significant improvements in their lung function.
- They had a reduced number of chest infections.
- They gained weight more quickly.
“These findings are groundbreaking. For the first time, we have shown that Kalydeco is safe and effective in newborns with CF. This is a major step forward in the treatment of CF.”
“This is a really important finding,” said David Ramsden, the Trust’s chief executive. “Kalydeco has already made a big difference to the lives of many people with CF, and now we know that it can also be safely and effectively used in newborns.”
ENDS
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Mehek 
Divya