Active Ingredient: Eculizumab
Eculizumab is indicated in adults and children for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Intravenous, 600 milligrams eculizumab, once weekly, over the duration of 4 weeks. Afterwards, intravenous, 900 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, intravenous, 900 milligrams eculizumab, once every 14 to 16 days.
The PNH dosing regimen for adult patients (≥18 years of age) consists of a 4-week initial phase followed by a maintenance phase:
aHUS patients should be monitored for signs and symptoms of thrombotic microangiopathy (TMA).
Eculizumab treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of eculizumab is clinically indicated.
Eculizumab may be administered to patients aged 65 years and over. There is no evidence to suggest that any special precautions are needed when older people are treated–although experience with eculizumab in this patient population is still limited.
The diluted solution of eculizumab should be administered by intravenous infusion over 25–45 minutes (35 minutes ± 10 minutes) in adults.
Patients should be monitored for one hour following infusion. If an adverse event occurs during the administration of eculizumab, the infusion may be slowed or stopped at the discretion of the physician. If the infusion is slowed, the total infusion time may not exceed two hours in adults and four hours in paediatric patients under 18 years of age.
There is limited safety data supporting home-based infusions, additional precautions in the home setting such as availability of emergency treatment of infusion reactions or anaphylaxis are recommended.
For:
Regimen A: In case that patient weight is ≥ 40 kg, intravenous, 600 milligrams eculizumab, once weekly, over the duration of 4 weeks. Afterwards, in case that patient weight is ≥ 40 kg, intravenous, 900 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 40 kg, intravenous, 900 milligrams eculizumab, once every 14 to 16 days.
Regimen B: In case that patient weight is ≥ 30 kg and patient weight is < 40 kg, intravenous, 600 milligrams eculizumab, once weekly, over the duration of 2 weeks. Afterwards, in case that patient weight is ≥ 30 kg and patient weight is < 40 kg, intravenous, 900 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 30 kg and patient weight is < 40 kg, intravenous, 900 milligrams eculizumab, once every 2 weeks.
Regimen C: In case that patient weight is ≥ 20 kg and patient weight is < 30 kg, intravenous, 600 milligrams eculizumab, once weekly, over the duration of 2 weeks. Afterwards, in case that patient weight is ≥ 20 kg and patient weight is < 30 kg, intravenous, 600 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 20 kg and patient weight is < 30 kg, intravenous, 600 milligrams eculizumab, once every 2 weeks.
Regimen D: In case that patient weight is ≥ 10 kg and patient weight is < 20 kg, intravenous, 600 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 10 kg and patient weight is < 20 kg, intravenous, 300 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 10 kg and patient weight is < 20 kg, intravenous, 300 milligrams eculizumab, once every 2 weeks.
Regimen E: In case that patient weight is ≥ 5 kg and patient weight is < 10 kg, intravenous, 300 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 5 kg and patient weight is < 10 kg, intravenous, 300 milligrams eculizumab, one dose, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 5 kg and patient weight is < 10 kg, intravenous, 300 milligrams eculizumab, once every 3 weeks.
Paediatric PNH patients with body weight ≥40 kg are treated with the adult dosing recommendations. The PNH dosing regimen consists of a 4-week initial phase followed by a maintenance phase:
In paediatric PNH patients with body weight below 40 kg, the eculizumab dosing regimen consists of:
| Patient Body Weight | Initial Phase | Maintenance Phase |
|---|---|---|
| 30 to <40 kg | 600 mg weekly for the first 2 weeks | 900 mg at week 3; then 900 mg every 2 weeks |
| 20 to <30 kg | 600 mg weekly for the first 2 weeks | 600 mg at week 3; then 600 mg every 2 weeks |
| 10 to <20 kg | 600 mg single dose at week 1 | 300 mg at week 2; then 300 mg every 2 weeks |
| 5 to <10 kg | 300 mg single dose at week 1 | 300 mg at week 2; then 300 mg every 3 weeks |
Eculizumab has not been studied in patients with PNH who weigh less than 40 kg. The posology of eculizumab to be used in paediatric patients with PNH patients weighing less than 40 kg is identical to the weight-based dose recommendation provided for paediatric patients with aHUS. Based on the pharmacokinetic (PK)/pharmacodynamic (PD) data available in patients with aHUS and PNH treated with eculizumab, this body-weight based dose regimen for paediatric patients is expected to result in an efficacy and safety profile similar to that in adults.
Eculizumab treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of Soliris is clinically indicated.
The diluted solution of eculizumab should be administered by intravenous infusion over 25–45 minutes (35 minutes ± 10 minutes) in adults.
Patients should be monitored for one hour following infusion. If an adverse event occurs during the administration of eculizumab, the infusion may be slowed or stopped at the discretion of the physician. If the infusion is slowed, the total infusion time may not exceed two hours in adults and four hours in paediatric patients under 18 years of age.
There is limited safety data supporting home-based infusions, additional precautions in the home setting such as availability of emergency treatment of infusion reactions or anaphylaxis are recommended.
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