Kalydeco is a medicine used to treat cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs. Kalydeco is used: * on its own, to treat cystic fibrosis in patients aged 1 month and above who weigh at least 3 kg and who have certain mutations (changes) in the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR). * together with a medicine containing tezacaftor / ivacaftor to treat patients aged 6 years and above who have inherited the *F508del* mutation in the *CFTR* gene from both parents or who have inherited the *F508del* mutation from one parent and have certain other mutations in the *CFTR* gene; * together with another medicine containing ivacaftor / tezacaftor / elexacaftor to treat patients aged 2 years and above who have at least one non\-class I mutation in the *CFTR* gene. Kalydeco contains the active substance ivacaftor.
Therapeutic Indication
Kalydeco tablets are indicated: \- As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. \- In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711\+3A→G, S945L, S977F, R1070W, D1152H, 2789\+5G→A, 3272 26A→G, and 3849\+10kbC→T. \- In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one non\-Class I mutation in the CFTR gene. Kalydeco granules are indicated: \- As monotherapy for the treatment of infants aged at least 1 month, toddlers and children weighing 3 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. \- In a combination regimen with ivacaftor/tezacaftor/elexacaftor 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 mutation in the CFTR gene.
Therapeutic Area (MeSH)
ATC Code
R07AX02
ATC Item
N/A
Pharmacotherapeutic Group
Other respiratory system products
Active Substance (Summary)
INN / Common Names
EMA Name
Kalydeco
Medicine Name
Kalydeco
Aliases
N/ANo risk management plan link.
