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 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 Kaftrio. The marketing authorisation holder for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.

The CHMP recommended the approval of two new presentations of Kaftrio (60mg/40mg/80mg and 75mg/50mg/100mg granules in sachet) for use in children aged 2 to 5 years of age.

For information, the indication for Kaftrio granules will be as follows:

Kaftrio granules are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 2 to less than 6 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

For information, the indication for Kaftrio tablets remains unchanged and is 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.

CHMP post-authorisation summary of positive opinion for Kaftrio (X-33)

AdoptedReference Number: EMA/344229/2023

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:
  14/09/2023
• Status:
  Positive