Ivacaftor

Chemical formula: C₂₄H₂₈N₂O₃  Molecular mass: 392.491 g/mol  PubChem compound: 16220172

Therapeutic indications

Ivacaftor is indicated for:

Cystic fibrosis

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 - 65 years old)

Ivacaftor is 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 F508del mutation in the CFTR gene.

For this indication, competent medicine agencies globally authorize below treatments (click for details):

Contraindications

Ivacaftor is contraindicated in the following cases:

Lactation

Lactation

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