1.1. Adults

Recommended doses for complement inhibitor as well as enzyme/protein replacement therapy FDA-approved for use in adults are summarized in Tables 1-3.  Patient profiles containing doses exceeding maximum recommendations will be reviewed.

Table 1. Adult Complement Inhibitor Maximum Dosages1-14
Drug Name Treatment Indication Dosage Form/Strength Maximum Recommended Dosage 
Berotralstat (Orladeyo®) Hereditary angioedema (HAE) prophylaxis 110 mg, 150 mg oral capsule 150 mg by mouth daily*
C1 esterase inhibitor, human (Berinert®) HAE treatment 500 international unit (IU) single-use vial for reconstitution 20 IU/kg by IV injection as a single dose
C1 esterase inhibitor, human (Cinryze®) HAE attacks, routine prevention 500 unit single-use vial for reconstitution 1000 U by IV infusion every 3-4 days+
C1 esterase inhibitor, human (Haegarda®) HAE attacks, routine prevention 2000, 3000 IU single-use vials for reconstitution 60 IU/kg subcutaneously twice weekly (every 3 to 4 days)
C1 esterase inhibitor, recombinant (Ruconest®) HAE treatment 2100 IU/14 mL single-use vial for reconstitution
  • Greater than or equal to  84 kg:
    • 4200 units as a single IV dose; may repeat x1 if attack symptoms persist in 24-hour period
  • Less than 84 kg:
    • 50 units/kg as single IV dose up to a maximum of 4200 units per dose; may repeat x1 if attack symptoms persist in 24-hour period
Ecallantide (Kalbitor®) HAE treatment 10 mg/ml single-use vials x 3 30 mg subcutaneously as three separate 10 mg injections; may repeat x1 in 24-hour period if attack symptoms persist
eculizumab (Soliris®) Atypical hemolytic uremic syndrome (aHUS) 300 mg/30 mL single-dose vial Initial 900 mg IV infusion once weekly for four weeks followed by a 1200 mg IV infusion one week later, maintenance 1200 mg IV infusion over 1-4 hours every two weeks thereafter 
  Myasthenia gravis, generalized   Initial 900 mg IV infusion once weekly for four weeks followed by a 1200 mg IV infusion one week later, maintenance 1200 mg IV infusion over 1-4 hours every two weeks
  Neuromyelitis optica spectrum disorder   Initial 900 mg IV infusion once weekly for four weeks followed by a 1200 mg IV infusion one week later, maintenance 1200 mg IV infusion over 1-4 hours every two weeks
  Paroxysmal nocturnal hemoglobinuria (PNH)   Initial 600 mg IV infusion once weekly for four weeks followed by a 900 mg IV infusion one week later, maintenance 900 mg IV infusion over 1-4 hours every two weeks thereafter
icatibant acetate (Firazyr®, Sajazir®) HAE treatment 30 mg/3 mL prefilled syringe, 30 mg/ 3 mL solution for injection 30 mg subcutaneously as single dose; may repeat x2 with 6 hours between doses in 24-hour period if attack symptoms persist (maximum 3 doses per 24-hour period) 
lanadelumab-flyo (Takhzyro®) HAE, routine prevention 300 mg/2 mL vial 300 mg subcutaneously every 2 weeks; every 4-week dosing possible in patients well-controlled for Greater than 6 months
pegcetacoplan (Empaveli®) PNH 1080 mg/ 20 mL solution for injection 1080 mg subcutaneously twice weekly
ravulizumab (Ultomiris®) aHUS^
PNH		 300 mg/3 mL, 100 mg/11 mL intravenous solution in single-dose vial
  • Greater than or equal to 100 kg:
    • 3600 mg as intravenous infusion every 8 weeks starting 2 weeks after loading dose (loading dose = 3000 mg)
  • 60-99 kg:
    • 3300 mg as intravenous infusion every 8 weeks starting 2 weeks after loading dose (loading dose = 2700 mg)
  • 40-59 kg:
    • 3000 mg as intravenous infusion every 8 weeks starting 2 weeks after loading dose (loading dose = 2400 mg)
  • 30-39 kg:
    • 2700 mg as intravenous infusion every 8 weeks starting 2 weeks after loading dose (loading dose = 1200 mg)
  • 20-29 kg:
    • 2100 mg as intravenous infusion every 8 weeks starting 2 weeks after loading dose (loading dose = 900 mg)
  • 10-19 kg:
    • 600 mg as intravenous infusion every 4 weeks starting 2 weeks after loading dose (loading dose = 600 mg)
  • 5-9 kg:
    • 300 mg as intravenous infusion every 4 weeks starting 2 weeks after loading dose (loading dose = 600 mg)

Legend

  • * Berotralstat is dosed at 110 mg by mouth daily in patients with moderate to severe hepatic impairment, chronic use of P-glycoprotein (P-gp) or BCRP (breast cancer resistance protein) inhibitors, or patients with persistent gastrointestinal (GI) reactions 
  • + in patients not responding adequately, doses up to 2500 units (not exceeding 100 u/kg) every 3 or 4 days may be utilized based on individual patient response
  • ^ treat atypical hemolytic-uremic syndrome with ravulizumab for at least six months
Table 2. Adult Enzyme Replacement Therapy Maximum Dosages1,2,15-29
Drug Name Treatment Indication Dosage Form/Strength Maximum Recommended Dosage
agalsidase beta (Fabrazyme®) Fabry disease 5 mg, 35 mg single-use vials 1 mg/kg by intravenous (IV) infusion every 2 weeks
alglucosidase alfa (Lumizyme®) Pompe disease (lysosomal acid alpha-glucosidase (GAA) deficiency) 50 mg single-use vial 20 mg/kg as IV infusion every 2 weeks
Avalglucosidase alfa (Nexviazyme®) Late onset Pompe disease (lysosomal acid alpha-glucosidase (GAA) deficiency) 100 mg single use vial
  • Greater than or equal to  30 kg:
    • 20 mg/kg as IV infusion every 2 weeks
  • Less than 30 kg:
    • 40 mg/ kg as IV infusion every 2 weeks
elapegademase (Revcovi®) Adenosine deaminase severe combined immunodeficiency 2.4 mg/1.5 mL intramuscular solution
  • treatment naïve:
    • 0.2 mg/kg as intramuscular injection twice weekly for a minimum of 12 to 24 weeks; titrate up by 0.033 mg/kg/week to maintain ADA trough Greater than 30 mmol/hr/L, trough deoxyadenosine nucleotides less than 0.02 mmol/L, and maintain immune reconstitution
  • patients transitioning from pegademase:
    • Patients receiving pegademase doses less than or equal to 30 U/kg should receive elapegademase 0.2 mg/kg intramuscularly once weekly; patients with pegademase doses Greater than 30 U/kg should receive equivalent elapegademase dose*
elosulfase alfa (Vimizim®) Mucopoly-saccharidosis (MPS) IVA (Morquio A syndrome) 5 mg/5 mL single-use vial 2 mg/kg by IV infusion once weekly
galsulfase (Naglazyme®) MPS VI (Maroteaux-Lamy syndrome) 5 mg/5 mL preservative-free vials 1 mg/kg by IV infusion once weekly
idursulfase (Elaprase®) MPS II (Hunter syndrome) 6 mg/3 mL single-use vial 0.5 mg/kg as IV infusion once weekly
imiglucerase (Cerezyme®) Gaucher disease, type 1 (non-neuropathic) 400 mg vials for reconstitution 60 U/kg by IV infusion over 1-2 hours every 2 weeks
laronidase (Aldurazyme®) MPS 1 (Hurler, Hurler-Scheie forms; Scheie form with moderate to severe symptoms) 2.9 mg/5 mL single-use vials 0.58 mg/kg by IV infusion once weekly
migalastat (Galafold®) Fabry disease with amenable galactosidase alpha gene (GLA) variant 123 mg oral capsule 123 mg by mouth every other day
sacrosidase (Sucraid®) Congenital sucrase-isomaltase deficiency (CSID) 8500 international units/mL as 118 mL oral solution bottles, 8500 units/ mL as 2 mL oral solution Greater than 15 kg: 17,000 units orally mixed in 2-4 ounces of water or milk with each meal or snack
sebelipase alfa (Kanuma®) Wolman Disease (Lysosomal acid lipase (LAL) deficiency) 20 mg/10 mL single-use vial 3 mg/kg by IV infusion once every other week
taliglucerase alfa (Elelyso®) Gaucher disease, type 1 200 unit single-use vials for reconstitution
  • treatment-naïve:
    • 60 U/kg by IV infusion once every 2 weeks
  • previously treated with imiglucerase:
    • use same unit/kg dosage for taliglucerase that was prescribed for imiglucerase and administer every two weeks
velaglucerase alfa (Vpriv®) Gaucher disease, type 1 400 unit single-use vials for reconstitution
  • treatment-naïve:
    • 60 U/kg as IV infusion every 2 weeks
  • previously treated with imiglucerase:
    • use same unit/kg dosage for velaglucerase that was prescribed for imiglucerase and administer every two weeks
vestronidase alfa-vjbk (Mepsevii®) MPS VII (Sly syndrome), excluding central nervous system symptoms 10 mg/5 mL single-use vial 4 mg/kg as IV infusion every two weeks

Legend

  • * elapegademase equivalent dose to pegademase: pegademase dose (U/kg) divided by 150
Table 3. Adult Protein Replacement Therapy Maximum Dosages1,2,30
Drug Name Treatment Indication Dosage Form/Strength Maximum Recommended Dosage
protein C concentrate (Ceprotin®) Severe congenital protein C deficiency (acute episode*) 500 IU, 1000 IU single-use vial for reconstitution 100-120 IU/kg initial dose by IV infusion, followed by 60-80 IU/kg every 6 hours for 3 doses by IV infusion
  Severe congenital protein C deficiency (short-term prophylaxis/ maintenance dose*)   45-60 IU/kg every 6 to 12 hours by IV infusion
  Severe congenital protein C deficiency (long-term prophylaxis*)   45-60 IU/kg every 12 hours by IV infusion

Legend

  • * maximum protein C concentrate infusion rate: 2 ml/min