Source: European Medicines Agency (EU) Revision Year: 2026 Publisher: Swedish Orphan Biovitrum AB (publ), SE-112 76 Stockholm, Sweden
ASPAVELI is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.
ASPAVELI is indicated for the treatment of adult and adolescent patients aged 12 to 17 years with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in combination with a renin-angiotensin system (RAS) inhibitor, unless RAS inhibitor treatment is not tolerated or contraindicated.
Therapy should be initiated under the supervision of a healthcare professional experienced in the management of patients with haematological or renal disorders. Self-administration and home infusion should be considered for patients who have tolerated treatment well in experienced treatment centres. The decision of a possibility of self-administration and home infusions should be made after evaluation and recommendation from the treating physician.
Pegcetacoplan can be given by a healthcare professional or administered by the patient or caregiver following proper instruction.
Pegcetacoplan is administered twice weekly as a 1 080 mg subcutaneous infusion with a commercially available syringe system infusion pump or on-body delivery system, that can deliver doses up to 20 mL. The twice weekly dose should be administered on Day 1 and Day 4 of each treatment week.
PNH is a chronic disease and treatment with ASPAVELI is recommended to continue for the patient's lifetime, unless the discontinuation of this medicinal product is clinically indicated (see section 4.4).
For the first 4 weeks, pegcetacoplan is administered as twice weekly subcutaneous doses of 1 080 mg in addition to the patient's current dose of C5 inhibitor treatment to minimise the risk of haemolysis with abrupt treatment discontinuation. After 4 weeks, the patient should discontinue C5 inhibitor before continuing on monotherapy with ASPAVELI.
Switches from complement inhibitors other than eculizumab have not been studied. Discontinuing other complement inhibitors before reaching steady-state of pegcetacoplan should be done with caution (see section 5.2).
The dosing regimen may be changed to 1 080 mg every third day (e.g., Day 1, Day 4, Day 7, Day 10, Day 13, and so forth) if a patient has a lactate dehydrogenase (LDH) level greater than 2 x upper limit of normal (ULN). In the event of a dose increase, LDH should be monitored twice weekly for at least 4 weeks (see section 4.4).
Pegcetacoplan is administered twice weekly as a subcutaneous infusion with a commercially available syringe system infusion pump or on-body delivery system, that can deliver doses up to 20 mL. The twice weekly dose should be administered on Day 1 and Day 4 of each treatment week.
C3G and primary IC-MPGN are chronic diseases. Discontinuation of this medicinal product is not recommended unless clinically indicated.
Pegcetacoplan is administered twice weekly as a 1 080 mg subcutaneous infusion.
For adolescent patients, the dosing regimen is based on the patient´s body weight and consists of the following:
| Body weight | First dose (infusion volume) | Second dose (infusion volume) | Maintenance dose (infusion volume) |
| ≥50 kg | 1 080 mg twice weekly (20 mL) | ||
| 35 to <50 kg | 648 mg (12 mL) | 810 mg (15 mL) | 810 mg twice weekly (15 mL) |
| 30 to <35 kg | 540 mg (10 mL) | 540 mg (10 mL) | 648 mg twice weekly (12 mL) |
If a dose of pegcetacoplan for treatment of PNH, C3G or primary IC-MPGN is missed, it should be administered as soon as possible, then the regular schedule should be resumed even if this results in an interval of less than 3 days between the replacement dose and the subsequent dose.
Diagnosis of post-transplant recurrent C3G or primary IC-MPGN should be made based on a renal allograft biopsy. C3G or primary IC-MPGN recurrence may be detected in a routine post-transplant biopsy; otherwise, a biopsy should be performed when clinical signs indicate recurrent disease. As done in study APL2-C3G-204 (see section 5.1), treatment with pegcetacoplan can be started before the onset of clinical signs such as estimated glomerular filtration rate (eGFR) decrease or urine to protein-to-creatine ratio (uPCR) increase. There is limited experience with the use of pegcetacoplan in patients with recurrent C3G or primary IC-MPGN after transplantation in clinical studies (see section 5.1).
Although there were no apparent age-related differences observed in clinical studies, the number of patients aged 65 and over is not sufficient to determine whether they respond differently from younger patients. There is no evidence indicating any special precautions are required for treating an elderly population.
Severe renal impairment (creatinine clearance <30 mL/min) had no effect on the pharmacokinetics (PK) of pegcetacoplan; therefore, pegcetacoplan dose adjustment in patients with renal impairment is not necessary. There are no data available for the use of pegcetacoplan in patients with end-stage renal disease (ESRD) requiring dialysis (see section 5.2).
The safety and efficacy of pegcetacoplan have not been studied in patients with hepatic impairment; however, no dose adjustment is recommended, as hepatic impairment is not expected to impact clearance of pegcetacoplan.
The safety and efficacy of ASPAVELI in children with PNH aged 0 to <18 years have not yet been established. No data are available.
The safety and efficacy of ASPAVELI in children with C3G or primary IC-MPGN aged below 12 years have not been established. No data are available.
This medicinal product should not be used in children <12 years of age, as non-clinical safety data are not available for this age group.
ASPAVELI should only be administered via subcutaneous administration using a commercially available syringe system infusion pump or on-body delivery system.
This medicinal product can be self-administered. When self-administration is initiated, the patient will be instructed by a qualified healthcare professional in infusion techniques, the use of a syringe system infusion pump or an on-body delivery system, the keeping of a treatment record, the recognition of possible adverse reactions, and measures to be taken in case these occur.
Infusion into areas where the skin is tender, bruised, red, or hard should be avoided. Infusion into tattoos, scars, or stretch marks should be avoided. The infusion should be started promptly after drawing this medicinal product into the syringe. Administration should be completed within 2 hours after preparing the syringe. For instructions on the preparation and infusion of the medicinal product, see section 6.6.
In the postmarketing setting, cases of overdose have been reported, with no new safety events observed. In case of overdose, it is recommended that the patient be monitored for any signs or symptoms of adverse reactions and appropriate symptomatic treatment be instituted.
30 months.
Store in a refrigerator (2°C–8°C).
Store in the original carton to protect from light.
A Type I glass vial with a stopper (chlorobutyl or bromobutyl), and a seal (aluminium) with a flip-off cap (polypropylene) containing 54 mg/mL of sterile solution.
Each single pack contains 1 vial.
Multipack containing 8 (8 packs of 1) vials.
Not all pack sizes may be marketed.
ASPAVELI comes as a ready-to-use solution in single-use vials. Because the solution contains no preservative, this medicinal product should be infused immediately after preparing the syringe.
ASPAVELI is a clear, colourless to slightly yellowish aqueous solution. Do not use if the liquid looks cloudy, contains particles, or is dark yellow.
Always bring the vial to the room temperature for approximately 30 minutes before use.
Remove the protective flip cap from the vial to expose the central portion of the gray rubber stopper of the vial. Clean the stopper with a new alcohol wipe and allow the stopper to dry. Do not use if the protective flip cap is missing or damaged.
Preparing the syringe:
Option 1: If using a needleless transfer device (such as a vial adapter), follow the instructions provided by the device manufacturer.
Option 2: If transfer is done using a transfer needle and a syringe, follow the instructions below:
Administration:
ASPAVELI should only be administered via subcutaneous administration using either a syringe system infusion pump or an on-body delivery system:
Any unused medicinal product or waste material should be disposed of in accordance with local requirements.
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