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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

Key facts

Agency product number
International non-proprietary name (INN) or common name
  • ivacaftor
Active substance
  • ivacaftor
Therapeutic area
Cystic Fibrosis
Accelerated assessment

This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

Date opinion adopted
Company name
Vertex Pharmaceuticals (Ireland) Limited
Application type

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