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1.2. Pediatrics

Tables 7 and 8 summarize ivacaftor dosing recommendations as monotherapy and combination therapy for pediatric patients. Ivacaftor is not recommended in patients less than one month of age1,2. Safety and efficacy of lumacaftor/ivacaftor combination therapy in children less than one year of age have not been established1,3,8. Tezacaftor/ivacaftor safety and efficacy have not been determined in children below 6 years of age, while elexacaftor/tezacaftor/ivacaftor efficacy/safety have not been established in children younger than two years of age1,4,5. Dosage adjustments for ivacaftor monotherapy and combination therapy when administered concomitantly with cytochrome P450 3A4 inhibitors are summarized in Table 9.

Table 7. Maximum Recommended Pediatric Ivacaftor Dosages: Monotherapy1-4
Drug NameTreatment IndicationDosage Form/StrengthMaximum Recommended Dosage
ivacaftor (Kalydeco)CF (patients with one mutation in CFTR gene responsive to ivacaftor potentiation)

5.8 mg, 13.4 mg, 25 mg, 50 mg, 75 mg oral granules

150 mg oral tablets

  • children/adolescents 6 years and older:
    • 150 mg orally every 12 hours with fat-containing food
  • infants/ children 6 months to less than 6 years (greater than 14 kg):
    • 75 mg as oral granules every 12 hours with fat-containing food
  • infants/children 6 months to less than 6 years (7 kg to less than 14 kg):
    • 50 mg as oral granules every 12 hours with fat-containing food
  • infants/children 6 months to less than 6 years (5 kg to less than 7 kg):
    • 25 mg as oral granules every 12 hours with fat-containing food
  • infants 4 months to less than 6 months weighing 5 kg or more:
    • 25 mg as oral granules every 12 hours with fat-containing food†
  • infants 2 months to less than 4 months weighing 3 kg or more:
    • 13.4 mg as oral granules every 12 hours with fat-containing food†*
  • infants 1 month to less than 2 months weighing 3 kg or more:
    • 5.8 mg as oral granules every 12 hours with fat-containing food†*

Legend:

  • CF = cystic fibrosis
  • CFTR = cystic fibrosis transmembrane conductance regulator
  • * = ivacaftor (Kalydeco) is not recommended for use in pediatric patients under 1 month of age
  • † = Use of ivacaftor (Kalydeco) in pediatric patients aged 1 year to less than 6 months born at a gestational age less than 37 weeks has not been evaluated
Table 8. Maximum Recommended Pediatric Ivacaftor Dosages: Combination Therapy1,3-5/sup>
Drug NameTreatment IndicationDosage Form/StrengthMaximum Recommended Dosage 
lumacaftor/ivacaftor (Orkambi)CF (patients homozygous for F508del mutation in the CFTR gene)

75 mg/94 mg, 100 mg/125 mg, 150 mg/188 mg oral granules

100 mg/125 mg, 200 mg/125 mg oral tablets

  • children/ adolescents 12 years and older:
    • 400 mg/250 mg (2 x 200 mg lumacaftor/125 mg ivacaftor tablets) orally every 12 hours with fat-containing food
  • children 6 to less than 12 years:
    • 200 mg/250 mg (2 x 100 mg lumacaftor/125 mg ivacaftor tablets) orally every 12 hours with fat-containing food
  • children 2-5 years (greater than or equal to 14 kg):
    • 150 mg/188 mg oral granules every 12 hours with fat-containing food
  • children 2-5 years (less than 14 kg):
    • 100 mg/125 mg oral granules every 12 hours with fat-containing food
  • children 1-2 years (greater than or equal to14 kg):
    • 150/188 mg oral granules every 12 hours with fat-containing food
  • children 1-2 years (9 to less than 14 kg):
    • 100/125 mg oral granules every 12 hours with fat-containing food
  • children 1-2 years (7 to less than 9 kg):
    • 75/94 mg oral granules every 12 hours with fat-containing food
tezacaftor/ivacaftor (Symdeko)CF (patients homozygous for F508del mutation or have at least one mutation in the CFTR gene responsive to tezacaftor/ivacaftor)

tezacaftor 50 mg/ivacaftor 75 mg oral tablet co-packaged with ivacaftor 75 mg oral tablets

tezacaftor 100 mg/ivacaftor 150 mg oral tablet co-packaged with ivacaftor 150 mg oral tablets

  • children/adolescents 12-17 years:
    • tezacaftor 100 mg/ivacaftor 150 mg in morning and ivacaftor 150 mg in evening approximately 12 hours apart with fat-containing food
  • children 6-11 years (greater than 30 kg):
    • tezacaftor 100 mg/ ivacaftor 150 mg in morning and ivacaftor 150 mg in evening approximately 12 hours apart with fat-containing food
  • children 6-11 years (less than 30 kg):
    • tezacaftor 50 mg/ ivacaftor 75 mg in morning and ivacaftor 75 mg in evening approximately 12 hours apart with fat-containing food
elexacaftor/tezacaftor/ivacaftor (Trikafta)CF (patients have at least one F508del mutation in the CFTR gene)

elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg oral tablet co-packaged with ivacaftor 150 mg oral tablet

elexacaftor 50 mg/ tezacaftor 25 mg/ ivacaftor 37.5 mg oral tablet co-packaged with ivacaftor 75 mg oral tablet

elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg oral granules co-packaged with ivacaftor 75 mg oral granules

elexacaftor 80 mg/tezacaftor 40 mg/ivacaftor 60 mg oral granules co-packaged with ivacaftor 59.5 mg oral granules

  • children/adolescents 12-17 years:
    • 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in morning and one ivacaftor 150 mg tablet in evening approximately 12 hours apart with fat-containing food
  • children 6-11 years greater than or equal to 30 kg):
    • 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning and one ivacaftor 150 mg tablet in the evening approximately 12 hours apart with fat-containing food
  • children 6-11 years (less than 30 kg):
    • 2 tablets (elexacaftor 50 mg/ tezacaftor 25 mg/ ivacaftor 37.5 mg) in the morning and one ivacaftor 75 mg tablet in the evening approximately 12 hours apart with fat-containing food

 Legend:

  • CF = cystic fibrosis
  • CFTR = cystic fibrosis transmembrane conductance regulator
Table 9. Ivacaftor Pediatric Dosage Adjustments with Concurrent CYP3A4 Inhibitor Therapy1-5
Ivacaftor Drug NameConcurrent CYP3A4 Inhibitor TherapyDosage Recommendations*
ivacaftor (Kalydeco)strong CYP3A inhibitors (e.g., ketoconazole)
  • Greater than or equal to 6 years of age:
    • 150 mg twice weekly
  • 6 months to less than 6 years of age:
    • Greater than or equal to 14 kg:
      • 75 mg granule packet twice weekly
    • 7 kg to less than 14 kg:
      • 50 mg granule packet twice weekly
    • 5 kg to less than 7 kg:
      • 25 mg granule packet twice weekly
  • less than 6 months of age:
    • Use not recommended
ivacaftor (Kalydeco)moderate CYP3A inhibitors (e.g., erythromycin, fluconazole)
  • Greater than 6 years of age:
    • 150 mg orally once daily
  • 6 months to less than 6 years of age:
    • Greater than or equal to 14 kg:
      • 75 mg granule packet orally once daily
    • 7 kg to less than 14 kg:
      • 50 mg granule packet orally once daily
    • 5 kg to less than 7 kg:
      • 25 mg granule packet orally once daily
  • less than 6 months of age:
    • Use not recommended
lumacaftor/ivacaftor (Orkambi)strong CYP3A inhibitors (e.g., ketoconazole)
  • Greater than or equal to 12 years:
    • lumacaftor/ivacaftor dosages should be reduced to 200 mg/125 mg once daily for the first week, followed by 2 tablets (lumacaftor 400 mg/ivacaftor 250 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 6 to 11 years:
    • lumacaftor/ivacaftor dosages should be reduced to 100 mg/125 mg once daily for the first week, followed by 2 tablets (lumacaftor 200 mg/ivacaftor 250 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 2 to 5 years (greater than or equal to 14 kg):
    • lumacaftor/ivacaftor dosages should be reduced to 1 granule packet (150 mg/188 mg) every other day for the first week, followed by 1 granule packet (lumacaftor 150 mg/ivacaftor 188 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 2 to 5 years (less than 14 kg):
    • lumacaftor/ivacaftor dosages should be reduced to 1 granule packet (100 mg/125 mg) every other day for the first week, followed by 1 granule packet (lumacaftor 100 mg/ivacaftor 125 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 1-2 years greater than or equal to 14 kg):
    • lumacaftor/ivacaftor dosages should be reduced to 1 granule packet (150 mg/188 mg) every other day for the first week, followed by 1 granule packet (lumacaftor 150 mg/ivacaftor 188 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 1-2 years (9 to less than 14 kg):
    • lumacaftor/ivacaftor dosages should be reduced to 1 granule packet (100 mg/125 mg) every other day for the first week, followed by 1 granule packet (lumacaftor 100 mg/ivacaftor 125 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
  • 1-2 years (7 to less than 9 kg):
    • lumacaftor/ivacaftor dosages should be reduced to 1 granule packet (75 mg/94 mg) every other day for the first week, followed by 1 granule packet (lumacaftor 75 mg/ivacaftor 94 mg) every 12 hours thereafter; no dosage adjustments are needed if CYP3A inhibitors are initiated in patients already taking lumacaftor/ivacaftor
lumacaftor/ivacaftor (Orkambi)moderate CYP3A inhibitors (e.g., erythromycin, fluconazole)no dosage adjustments recommended
tezacaftor/ ivacaftor (Symdeko)strong CYP3A inhibitors (e.g., ketoconazole)
  • greater than or equal to 6 years to less than 12 years (greater than or equal to 30 kg) & patients greater than or equal to12 years:
    • Day 1: tezacaftor 100 mg/ivacaftor 150 mg in the morning
    • Days 2 and 3: no dosages administered
    • Day 4: tezacaftor 100 mg/ivacaftor 150 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue twice weekly (morning) administration schedule, with 3-4 days between dosages
  • greater than or equal to6 years to less than 12 years (less than 30 kg):
    • Day 1: tezacaftor 50 mg/ivacaftor 75 mg in the morning
    • Days 2 and 3: no dosages administered
    • Day 4: tezacaftor 50 mg/ivacaftor 75 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue twice weekly (morning) administration schedule, with 3-4 days between dosages
tezacaftor/ivacaftor (Symdeko)moderate CYP3A inhibitors (e.g., erythromycin, fluconazole)
  • greater than or equal to 6 years to less than 12 years (greater than or equal to30 kg) and patients greater than or equal to12 years:
    • Day 1: tezacaftor 100 mg/ivacaftor 150 mg in the morning
    • Day 2: ivacaftor 150 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule
  • greater than or equal to 6 years to less than 12 years (less than 30 kg):
    • Day 1: tezacaftor 50 mg/ivacaftor 75 mg in the morning
    • Day 2: ivacaftor 75 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule
elexacaftor/tezacaftor/ivacaftor (Trikafta)strong CYP3A inhibitors (e.g., ketoconazole)
  • greater than or equal to12 years of age:
    • 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning twice weekly, with 3-4 days between dosages
      The evening ivacaftor dose should NOT be given
  • 6-11 years of age:
    • weight greater than or equal to30 kg:
    • 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning twice weekly, with 3-4 days between doses 
      The evening ivacaftor dose should NOT be given
  • 6-11 years of age:
    • weight less than 30 kg:
    • 2 tablets (elexacaftor 50 mg/ tezacaftor 25 mg/ ivacaftor 37.5 mg) in the morning twice weekly, with 3-4 days between doses
      The evening ivacaftor dose should NOT be given
  • 2-5 years of age:
    • weight greater than or equal to14 kg:
    • one packet (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning twice weekly, with 3-4 days between doses
      The evening ivacaftor dose should NOT be given
  • 2-5 years of age:
    • weight less than 14 kg:
    • one packet (elexacaftor 80 mg/ tezacaftor 40 mg/ ivacaftor 60 mg) in the morning twice weekly, with 3-4 days between doses
      The evening ivacaftor dose should NOT be given
elexacaftor/tezacaftor/ivacaftor (Trikafta)moderate CYP3A inhibitors (e.g., erythromycin, fluconazole)
  • greater than or equal to 12 years of age:
    • Day 1: 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning
    • Day 2: ivacaftor 150 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule
  • 6-11 years of age:
    • Weight greater than or equal to 30 kg:
      • Day 1: 2 tablets (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning
      • Day 2: ivacaftor 150 mg in the morning
  • The evening ivacaftor dose should NOT be given
  • Continue alternate day (morning) administration schedule
  • 6-11 years of age:
    • Weight less than 30 kg:
      • Day 1: 2 tablets (elexacaftor 50 mg/ tezacaftor 25 mg/ ivacaftor 37.5 mg) in the morning
      • Day 2: ivacaftor 75 mg in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule
  • 2-5 years of age:
    • weight greater than or equal to 14 kg:
      • Day 1: one packet (elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg) in the morning
      • Day 2: one packet (ivacaftor 75 mg) in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule
  • 2-5 years of age:
    • weight less than 14 kg:
      • Day 1: one packet (elexacaftor 80 mg/ tezacaftor 40 mg/ ivacaftor 60 mg) in the morning 
      • Day 2: one packet (ivacaftor 59.5 mg) in the morning
    • The evening ivacaftor dose should NOT be given
    • Continue alternate day (morning) administration schedule

Legend:

  • CYP3A = cytochrome P450 3A