European CHMP recommends approval of wider use of ivacaftor in patients with cystic fibrosis and an R117H mutation in the CFTR gene

When there is an R117H CFTR mutation present, Kalydeco tablets are currently licensed only for use in adults. The CHMP has issued a positive opinion recommending this be extended to include adolescents and children aged 6 years and older and weighing 25 kg or more.

SPS commentary:

The CHMP has also recommended that Kalydeco granules be approved for use in the treatment of infants aged at least 6 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis who have an R117H CFTR mutation (currently not licensed for use in patients with this mutation).


European Medicines Agency