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C3 complement inhibitor (pegylated peptide) Pregnancy: §4.6, verbatim: 'There is a limited amount of data from the use of pegcetacoplan in pregnant women. Studies in animals have shown reproductive toxicity. Pegcetacoplan is not recommended during pregnancy and in women of childbearing potential not using contraception.' Contraception, verbatim: 'It is recommended that women of childbearing potential use effective contraception methods to prevent pregnancy during treatment with pegcetacoplan and for at least 8 weeks after the last dose.' Breast-feeding, verbatim: 'It is recommended to discontinue breast-feeding during pegcetacoplan treatment.'

Pegcetacoplan

Brand names: Aspaveli, Empaveli

Pegcetacoplan is a pegylated complement C3 inhibitor used to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: 1080 mg by subcutaneous infusion. §4.2, verbatim: 'Pegcetacoplan is administered twice weekly as a 1080 mg subcutaneous infusion with a commercially available syringe system infusion pump or on-body delivery system, that can deliver doses up to 20 mL.'
Route: Subcutaneous infusion by syringe-system infusion pump or on-body delivery system. §4.2, verbatim: 'When using a syringe system infusion pump, it should be infused in the abdomen, thighs, hips, or upper arms. Infusion sites should be at least 7.5 cm apart from each other. The infusion sites should be rotated between administrations. The infusion time is approximately 30 minutes (if using two sites) or approximately 60 minutes (if using one site).' With an on-body delivery system the site is the abdomen and 'the infusion time varies by patient and typically ranges from 30 to 60 minutes'. 'Administration should be completed within 2 hours after preparing the syringe.'
Frequency: Twice weekly. §4.2, verbatim: 'The twice weekly dose should be administered on Day 1 and Day 4 of each treatment week.' Escalation for breakthrough haemolysis, verbatim: 'The dosing regimen may be changed to 1080 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).'
maxDose is blank because §4.2 states no ceiling — the every-third-day schedule is the highest frequency described and carries no stated maximum dose. Duration, verbatim: 'Treatment with pegcetacoplan is recommended to continue for the patient's lifetime, unless the discontinuation of this medicinal product is clinically indicated.' Switching from a C5 inhibitor requires 4 weeks of OVERLAP, verbatim: 'For the first 4 weeks, pegcetacoplan is administered as twice weekly subcutaneous doses of 1080 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 this medicinal product. Switches from complement inhibitors other than eculizumab have not been studied.' Missed dose, verbatim: 'If a dose of pegcetacoplan ... 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.' Initiation is specialist-only, verbatim: 'Therapy should be initiated under the supervision of a healthcare professional experienced in the management of patients with haematological or renal disorders.' Vaccination against encapsulated bacteria at least 2 weeks before the first dose is mandatory (§4.3, §4.4). Elderly: 'There is no evidence indicating any special precautions are required for treating an elderly population.'

Paediatric dose

Route: Not applicable
Frequency: Not applicable
NO PAEDIATRIC PNH DOSE. §4.2, Paediatric population, verbatim: 'The safety and efficacy of ASPAVELI in children with PNH aged 0 to <18 years have not yet been established. No data are available.' dosePerKg is null and no weight-based calculation should be offered. The same SPC DOES give an adolescent regimen for C3G and primary IC-MPGN ('The safety and efficacy of ASPAVELI in children with C3G or primary IC-MPGN aged below 12 years have not been established'), weight-banded as 1080 mg twice weekly at 50 kg and above, 648 mg then 810 mg then 810 mg twice weekly at 35 to <50 kg, and 540 mg then 540 mg then 648 mg twice weekly at 30 to <35 kg — but those are RENAL indications, not this page's PNH indication, and must not be transferred here. If a child or adolescent needs complement inhibition, verify the indication, agent and dose against a children's formulary and specialist haematology or nephrology advice.

Dose adjustments

Renal

§4.2, verbatim: 'Dose adjustment in patients with severe renal impairment (creatinine clearance <30 mL/min) is not necessary. There are no data available for the use of pegcetacoplan in patients with end-stage renal disease (ESRD) requiring dialysis.'

Hepatic

§4.2, verbatim: '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.'

Dose auto-extracted from UK Summary of Product Characteristics (SPC) via the eMC; US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

  • Hypersensitivity to the active substance or to any of the excipients (§4.3)
  • Unresolved infection caused by encapsulated bacteria including Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae — therapy must not be initiated (§4.3)
  • Patients not currently vaccinated against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae — therapy must not be initiated 'unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination' (§4.3)

Side effects

  • Injection site reactions — in PNH: erythema (29%), pruritus (13%), swelling (12%), bruising (10%), pain (8%); infusion site reactions in C3G/IC-MPGN (33%) (§4.8)
  • Infections — upper respiratory tract infection (26% in PNH, very common in both populations), urinary tract infection, influenza; sepsis and opportunistic infections reported as common in PNH (§4.8)
  • Haemolysis (19% in PNH) — listed as one of the most serious adverse reactions (§4.8)
  • Abdominal pain (19%), diarrhoea (18%) (§4.8)
  • Headache (18%) and dizziness (10%) (§4.8)
  • Fatigue (15%) and pyrexia (14%) (§4.8)
  • Cough (11%), vaccination complication (11%), pain in extremity (12%), arthralgia (10%), back pain (8%) (§4.8)
  • Thrombocytopenia and neutropenia — common (§4.8)
  • Hypersensitivity reaction — very common; anaphylactic reaction and anaphylactic shock — uncommon (§4.8)
  • Hypokalaemia — common; hypertension — common (§4.8)
  • Acute kidney injury (13%) and pneumonia (9%) — the most serious adverse reactions in C3G or primary IC-MPGN (§4.8)

Monitoring

  • LDH and signs and symptoms of haemolysis — §4.4, verbatim: 'Patients with PNH receiving pegcetacoplan should be monitored regularly for signs and symptoms of haemolysis, including measuring LDH levels, and may require dose adjustment within the recommended dosing schedule.'
  • After a dose increase to every third day, LDH twice weekly for at least 4 weeks (§4.2)
  • Early signs of infection by encapsulated bacteria — §4.4, verbatim: 'All patients should be monitored for early signs of infections caused by encapsulated bacteria ... evaluated immediately if infection is suspected, and treated with appropriate antibiotics if necessary.'
  • Vaccination status — cover against Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y and B, and Haemophilus influenzae type B within 2 years before starting, or given at least 2 weeks before the first dose (§4.4)
  • Watch for hypersensitivity — discontinue the infusion immediately for a severe reaction including anaphylaxis (§4.4)
  • Injection site reactions and injection technique (§4.4)
  • Interpret aPTT with caution — §4.4: silica reagents in coagulation panels may interact with pegcetacoplan and artificially prolong aPTT

Clinical monograph

How it works

It binds complement protein C3 and its activation fragment C3b, providing broad control of the complement cascade and limiting both intravascular and extravascular haemolysis of PNH red cells.

Prescribing in practice

  • Because complement inhibition increases susceptibility to encapsulated organisms, vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type b is required before starting, with antibiotic cover if treatment cannot await vaccination.
  • On switching from a C5 inhibitor, regimens overlap to reduce the risk of haemolysis, and abrupt discontinuation can precipitate haemolysis requiring close monitoring.
  • It is given by subcutaneous infusion, typically via a pump, in accordance with the SPC.

Monitoring

Monitor haemoglobin, lactate dehydrogenase, reticulocytes and signs of haemolysis, and remain vigilant for symptoms of serious infection both during treatment and after any interruption.

Counselling the patient

  • Stress the importance of staying up to date with the required vaccinations and carrying a patient safety card.
  • Advise seeking urgent medical attention for fever or other features suggesting serious infection, and not stopping treatment without specialist advice.

Evidence & guidelines

The PEGASUS trial showed pegcetacoplan improved haemoglobin compared with eculizumab in paroxysmal nocturnal haemoglobinuria.

Reference: NICE TA778; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

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