Kaftrio - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation.

ivacaftor / tezacaftor / elexacaftor

Opinion

On 27 February 2025, 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 Kaftrio. The marketing authorisation holder for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.

The CHMP adopted extensions to the existing indications for Kaftrio film-coated tablets and granules in sachets to extend their use to patients with at least one non-class I CFTR mutation. The full indications for Kaftrio will therefore be as follows:¹

Kaftrio tablets are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one non-class I F508delmutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see sections4.2 and 5.1).

Kaftrio granules are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one non-class I F508delmutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see sections4.2 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 on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

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¹New text in bold, removed text as strikethrough

CHMP post-authorisation summary of positive opinion for Kaftrio WS-2551

AdoptedReference Number: EMA/CHMP/62074/2025

Key facts

• Name of medicine:
  Kaftrio
• EMA product number:
  EMEA/H/C/005269
• Active substance:
  • ivacaftor
  • tezacaftor
  • elexacaftor
• International non-proprietary name (INN) or common name:
  • ivacaftor
  • tezacaftor
  • elexacaftor
• Therapeutic area (MeSH):
  Cystic Fibrosis
• Anatomical therapeutical chemical (ATC) code:
  R07AX32
• Additional monitoring:

  This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

• Orphan:

  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.

• Marketing authorisation holder:
  Vertex Pharmaceuticals (Ireland) Limited
• Date of opinion:
  27/02/2025
• Status:
  Positive