Kalydeco

On 14 September 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Kalydeco. The marketing authorisation holder for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.

The CHMP adopted a new indication for Kalydeco granules to include treatment of cystic fibrosis (CF) in children aged between 2 and 6 years in a combination regimen with ivacaftor/tezacaftor/elexacaftor. For information, the full indications for Kalydeco granules will be as follows¹:

• As monotherapy for the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
• In a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one F508del mutation in the CFTR gene.

For information, the indications for Kalydeco tablets remain unchanged and are provided in the summary of product characteristics (SmPC).

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR) and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

¹New text in bold