Kaftrio

ivacaftor / tezacaftor / elexacaftor

Table of contents

Opinion

On 25 March 2021, 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 an extension to the existing indication to extend the use in all patients with at least one F508del mutation.

For information, the full indication for Kaftrio will be as follows: 1

Kaftrio is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.

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, removed text as strikethrough

Key facts

Name
Kaftrio
Agency product number
EMEA/H/C/005269
International non-proprietary name (INN) or common name
  • ivacaftor
  • tezacaftor
  • elexacaftor
Active substance
  • ivacaftor
  • tezacaftor
  • elexacaftor
Therapeutic area
Cystic Fibrosis
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.

Date opinion adopted
25/03/2021
Company name
Vertex Pharmaceuticals (Ireland) Limited
Status
Positive
Application type
Post-authorisation

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