Section 1: What Kaftrio is and what is it used for

Indication changed to: Kaftrio taken with ivacaftor is for patients aged 12 years and older who have CF, with at least one F508del mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene.”

4.1 Therapeutic Indications

Updated indication to “Kaftrio is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulatory (CFTR) gene.”

4.2 Posology and method of administration

Updated to match indication

Section 4.4 Special warnings and precautions for use: Elderly Population

Updated information to indicate studies did not include patients over 65 years old

Section 4.8: Undesirable effects: Summary of safety profile

Updated with study 445-104

Section 4.8: Undesirable effects: Paediatric population

Updated number of patients in safety data

5.1: Pharmacodynamic properties

Updated to include information on non-F508del CFTR variants

5.1: Pharmacodynamic properties- Effects on sweat chloride

Added Study 445-104 information and data

5.1: Pharmacodynamic properties- Clinical efficacy and safety

Added efficacy data for patients homozygous for F508del mutation or heterozygous for F508del mutation and a mutation with MF, a gating defect, or residual CFTR activity on the second allele

Study 445-104 added

Table 6: Primary and secondary efficacy analyses, full analysis set (study 445-104) added

Update to include local representative details for MAH holder in the PIL.

Initial Marketing Authorisation: 21 August 2020

Initial Marketing Authorisation: 21 August 2020