Pegcetacoplan
Also known as: APL-2, APL‑2, Aspaveli, Empaveli, POT-4 successor / compstatin analog, Syfovre
Source Pegcetacoplan at Peptiology
Save 10% with code PEPTI-BOSSRABBIT-10
Tapping Shop Now & Save 10% copies your 10% off code PEPTI-BOSSRABBIT-10 to your clipboard. Paste it at the Peptiology checkout to claim the discount.
Summary
Pegcetacoplan is a PEGylated cyclic peptide (compstatin analog) that inhibits complement C3. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Mechanism of Action
Binds complement component C3 and blocks its cleavage, thereby inhibiting the downstream complement cascade.
Routes of Administration
Goals & Uses
- Treatment of complement-mediated hemolytic anemiasHematologyModerate
- Reduction of extravascular hemolysis in PNHHematologyHigh
- Treatment of paroxysmal nocturnal hemoglobinuria (PNH)Hematology / Rare DiseaseHigh
- Treatment of geographic atrophy (GA) in AMDOphthalmologyHigh
- Paroxysmal nocturnal hemoglobinuriaHematologyHigh
Contraindications
- Unresolved Neisseria meningitidis infectionInfectious DiseaseHigh
- Hypersensitivity to pegcetacoplan or excipientsAllergy/ImmunologyHigh
- Hypersensitivity to pegcetacoplan or PEGHypersensitivityModerate
- Unvaccinated patients (encapsulated organisms)Infectious DiseaseHigh
Adverse Effects
- Infusion-related reactions (intravitreal: conjunctival hemorrhage, floaters)OphthalmologicCommon
- Serious infections (encapsulated bacteria)Infectious DiseaseUncommon
- Injection site reactionsLocalCommon
- HeadacheNeurologicCommonPain in the head or upper neck
- Hemolysis upon discontinuationHematologicUncommon
- FatigueGeneralCommonLow energy or tiredness
- InfectionsImmunologicCommon
- Diarrhea / gastrointestinal symptomsGastrointestinalCommon
- DiarrheaGastrointestinalUncommonLoose or frequent stools
Drug Interactions
- Live attenuated vaccinesModerate
- Vaccines (live attenuated)Moderate
- Eculizumab / RavulizumabModerate
Population Constraints
- PregnancyReproductive SafetyRelative
- Severe renal impairmentOrgan ImpairmentRelative
- Pediatric patientsAgeRelative
- Patients with active systemic infectionsInfectious DiseaseRelative
- BreastfeedingReproductiveRelative
Regulatory Status
- European UnionApprovedApproved: Paroxysmal nocturnal hemoglobinuriaApproved in 2022.
- United StatesApprovedApproved: Paroxysmal nocturnal hemoglobinuriaApproved in May 2021 (Empaveli).
- United KingdomApprovedApproved: Paroxysmal nocturnal hemoglobinuria (PNH) in adultsMHRA approved following EMA decision; NICE technology appraisal conducted.
First FDA‑approved C3 inhibitor (Empaveli).
Evidence & Sources
No sources recorded yet.
Frequently Asked Questions
What is Pegcetacoplan?
Pegcetacoplan is a PEGylated cyclic peptide (compstatin analog) that inhibits complement C3. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
What is Pegcetacoplan used for?
Pegcetacoplan is educationally associated with: Treatment of complement-mediated hemolytic anemias, Reduction of extravascular hemolysis in PNH, Treatment of paroxysmal nocturnal hemoglobinuria (PNH), Treatment of geographic atrophy (GA) in AMD, Paroxysmal nocturnal hemoglobinuria. Educational only — not medical advice.
How is Pegcetacoplan administered?
Recorded routes of administration: Intravitreal, Subcutaneous.
What are the potential side effects of Pegcetacoplan?
Reported adverse effects include: Infusion-related reactions (intravitreal: conjunctival hemorrhage, floaters), Serious infections (encapsulated bacteria), Injection site reactions, Headache, Hemolysis upon discontinuation, Fatigue, Infections, Diarrhea / gastrointestinal symptoms, Diarrhea. This list is not exhaustive — consult a qualified clinician.
Who should avoid Pegcetacoplan?
Recorded contraindications: Unresolved Neisseria meningitidis infection, Hypersensitivity to pegcetacoplan or excipients, Hypersensitivity to pegcetacoplan or PEG, Unvaccinated patients (encapsulated organisms). Consult a qualified clinician before use.