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On 17 October 2019, 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 recommended approval of a new 25-mg strength, for Kalydeco granules, and an extension to the existing indication to allow use in infants from 6 months of age and weighing from 5 kg to less than 25 kg.   

For information, the full indication for Kalydeco granules will be as follows:*

'Kalydeco granules are indicated for the treatment of children with cystic fibrosis (CF)infants aged 12 at least 6 months, toddlers and older and children weighing 75 kg to less than 25 kg with cystic fibrosis (CF) who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).'

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.

*New text in bold, deleted text in strikethrough

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.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

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

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